StemCell Therapy

Gene therapy has been studied for more than 40 years and can help stop or slow the effects of disease on the most basic level of the human bodyour genes. And to understand how it works, well start at the basics.

Genes are made up of DNA, which are blueprints to build enzymes and proteins that make our body work. As far as we know, humans have between 20,000 and 25,000 genes. We typically get two copies of each gene from our parents. They influence everything from the color of our hair to our immune system, but genes arent always built correctly. A small adjustment to them can change how our proteins work, which then alter the way we breathe, walk or even digest food. Genes can change as they go through inherited mutations, as they age, or by being altered or damaged by chemicals and radiation.

In the case that a gene changesalso known as mutatingin a way that causes disease, gene therapy may be able to help. Gene therapy is the introduction, removal or change in genetic materialspecifically DNA or RNAinto the cells of a patient to treat a specific disease. The transferred genetic material changes how a proteinor group of proteinsis produced by the cell.

This new genetic material or working gene is delivered into the cell by using a vector. Typically, viruses are used as vectors because they have evolved to be very good at sneaking into and infecting cells. But in this case, their motive is to insert the new genes into the cell. Some types of viruses being used are typically not known to cause disease and other times the viral genes known to cause disease are removed. Regardless of the type, all viral vectors are tested many times for safety prior to being used. The vector can either be delivered outside the body (ex-vivo treatment) or the vectors can be injected into the body (in-vivo treatment).

Other types of drugs are typically used to manage disease or infection symptoms to relieve pain, while gene therapy targets the cause of the disease. It is not provided in the form of a pill, inhalation or surgery, it is provided through an injection or IV.

What Counts as a Rare Disease?

Gene therapy treats diseases in patients that are rare and often life threatening. Rare is defined as any disease or disorder affecting fewer than 200,000 people in the U.S. by the National Institutes of Health. As of now, there are around 7,000 rare diseases, affecting a total of approximately one in ten people. Many of these rare diseases are caused by a simple genetic mutation inherited from one or both parents.

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Which Diseases Have Gene Therapies?

Of gene therapies up for approval over the next five years, 45 percent are anticipated to focus on cancer treatments and 38 percent are expected to treat rare inherited genetic disorders. Gene therapy can help add to or change non-functioning genescreating a great opportunity to assist with rare inherited disorders, which are passed along from parents. The mutation might be present on one or both chromosomes passed along to the children. The majority of gene therapies are currently being studied in clinical trials.

Some of these inherited diseases include (but are not limited to):

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Why Do We Use Viral Vectors?

As you know from cold and flu season, viruses are quite skilled in the art of invading our bodiesadding their genetic material into our cells. However, researchers have learned to harness this sneaky ability to our advantage. Viruses are often used as a vehicle to deliver good genes into our cells, as opposed to the ones that cause disease.

Viruses are sometimes modified into vectors as researchers remove disease-causing material and add the correct genetic material. In gene therapy, researchers often use adeno-associated viruses (AAV) as vectors. AAV is a small virus that isnt typically known to cause disease in the first place, significantly reducing a chance of a negative reaction.

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Gene Therapy Basics | Education | ASGCT American Society ...

Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.

A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can't cause disease when used in people. Some types of virus, such as retroviruses, integrate their genetic material (including the new gene) into a chromosome in the human cell. Other viruses, such as adenoviruses, introduce their DNA into the nucleus of the cell, but the DNA is not integrated into a chromosome.

The vector can be injected or given intravenously (by IV) directly into a specific tissue in the body, where it is taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector in a laboratory setting. The cells containing the vector are then returned to the patient. If the treatment is successful, the new gene delivered by the vector will make a functioning protein.

Researchers must overcome many technical challenges before gene therapy will be a practical approach to treating disease. For example, scientists must find better ways to deliver genes and target them to particular cells. They must also ensure that new genes are precisely controlled by the body.

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How does gene therapy work? - Genetics Home Reference - NIH

Nearly 20 years ago, scientists stunned the world when they announced they had decoded the genes that make up a human being. They hoped to use that genetic blueprint to advance something called gene therapy which locates and fixes the genes responsible for different diseases.

Now, a clinical trial at the National Institutes of Health is doing exactly that in an attempt to cure sickle cell anemia, a devastating genetic disease that kills hundreds of thousands of people around the world every year.

For the past 15 months we've been following the scientists, and patients, who are ushering in a genetic revolution.

Jennelle Stephenson: I'm excited.

Ray Stephenson Today is the big day.

It's the day after Christmas, 2017, and 27-year-old Jennelle Stephenson has come with her father and brother from Florida to the National Institutes of Health, just outside Washington, D.C.

Jennelle Stephenson: Good morning.

Dr. John Tisdale: Good morning.

She's one of a small group of patients to receive an infusion containing altered DNA.

Nurse: This is what they look like.

Jennelle Stephenson: Merry Christmas to me.

Brother: Best Christmas present ever.

Jennelle Stephenson: Yay.

The clear liquid in the bag contains Jennelle's stem cells that have been genetically modified.

Dr. John Tisdale: There are about 500 million in there.

Jennelle Stephenson: Oh, my goodness.

The hope is the new DNA in the cells will cure Jennelle of sickle cell anemia, a brutal disease that causes debilitating pain.

Dr. Jon LaPook: At its worst, on a scale of zero to 10, how bad was your pain?

Jennelle Stephenson: We can go beyond a 10. It's terrible, it's horrible.

Dr. Jon LaPook: Pain where?

Jennelle Stephenson: Everywhere. My back, my shoulders, elbows, arms, legs, even my cheekbones, just pain.

Dr. Jon LaPook: Can you actually describe it?

Jennelle Stephenson: It's a very sharp, like, stabbing, almost feels like bone-crushing pain. Feels like someone's kind of constricting your bones, and then releasing constantly.

Pain from sickle cell can occur anywhere blood circulates. That's because red blood cells, normally donut-shaped, bend into an inflexible sickle shape, causing them to pile up inside blood vessels. The resulting traffic jam prevents the normal delivery of oxygen throughout the body, leading to problems that include bone deterioration, strokes and organ failure.

The gene that causes sickle cell anemia evolved in places like sub-Saharan Africa because it protects people from malaria. There, millions have the disease, and it's estimated more than 50 percent of babies born with it die before the age of five.

In the United States, it affects a hundred thousand people, mostly African-Americans.

For Jennelle, having the disease as a child often meant spending Christmas in the hospital. As an adult, she struggled through pain to complete college, but keeping a job was tough because something as simple as walking up stairs could trigger "a pain crisis."

Dr. Jon LaPook: Do you have friends who've died from sickle cell?

Jennelle Stephenson: I do. Yes, younger than me.

Dr. Jon LaPook: And you've known this your whole life growing up?

Jennelle Stephenson: Right.

Dr. Jon LaPook: That you could potentially die early?

Jennelle Stephenson: Right. Yes.

Dr. Jon LaPook: Did you think you would die early?

Jennelle Stephenson: I did, actually. When I hit about 22, I was like, "You know, I'm-- for a sickle celler, I'm kind of middle-aged right now."

Dr. Jon LaPook: What are some of the things that you've always wanted to do that you couldn't do?

Jennelle Stephenson: Honestly, everybody laughs at me for this, I just want to run, to be honest.

Dr. Jon LaPook: Things that most people would take for granted.

Jennelle Stephenson: Just basic things.

One of the most cruel parts of the disease, Jennelle and other patients have told us, is being accused of faking pain to get narcotics, being labeled a "drug-seeker." During one trip to the emergency department, when she fell to the floor in pain, a doctor refused to help her.

Jennelle Stephenson: And I'm looking up at her, and I'm in tears, and, I'm like, "I'm doing the best that I can."

Dr. Jon LaPook: And you gotta be thinking.

Jennelle Stephenson: I just, sometimes I don't understand, I don't get it. Like... Sorry. I'm in so much pain, and you think I just want some morphine. And it just makes me sad that some people in the medical community just don't get it.

Dr. Francis Collins is director of the National Institutes of Health, the largest biomedical research agency in the world. He oversees a nearly 40 billion dollar budget that funds more than 400,000 researchers world-wide.

Dr. Collins was head of the Human Genome Project at the NIH in 2000 when he made a landmark announcement: after a decade of work, scientists had finally decoded the genes that make up a human being.

Dr. Jon LaPook: When did it all start for you?

Dr. Francis Collins: I got excited about genetics as a first-year medical student. A pediatric geneticist came to teach us about how genetics was relevant to medicine. And he brought patients to class and one of the first patients he brought was a young man with sickle cell disease who talked about the experience of sickle cell crises and how incredibly painful those are. And yet, it was all because of one single letter in the DNA that is misplaced, a "T" that should have been an "A." And that was profound. You could have all of that happen because of one letter that was misspelled.

The double helix of DNA is made up of billions of pieces of genetic information. What Dr. Collins is saying is, out of all that, it's just one error in the DNA code -- a "T" that should have been an "A" -- that causes sickle cell anemia. Fix that error, and you cure the disease.

But figuring out how to do that would take more than 20 years of research and a little serendipity.

Dr. Collins was playing in the NIH rock band in 2016 when his bass player -- hematologist Dr. John Tisdale -- started riffing on an idea.

Dr. John Tisdale: We'd finished setting up and went for a pizza before--

Dr. Francis Collins: I remember that.

Dr. John Tisdale: --before the gig. And at this point I pitched to Francis that it was really time that we do something definitive for sickle cell disease.

In the laboratory, Dr. Tisdale and his collaborators created a gene with the correct spelling. Then, to get that gene into the patient, they used something with a frightening reputation: HIV, the virus that causes AIDS. It turns out HIV is especially good at transferring DNA into cells.

Here's how it works. The corrected gene, seen here in yellow, is inserted into the HIV virus. Then, bone marrow stem cells are taken from of a patient with sickle cell anemia. In the laboratory those cells are combined with the virus carrying that new DNA.

Dr. John Tisdale: This virus will then find its way to one of those cells and drop off a copy or two of the correctly spelled gene. And then these cells will go back to the patient.

If the process works, the stem cells with the correct DNA will start producing healthy red blood cells.

Dr. Jon LaPook: I can hear people, our viewers out there, thinking, "Wait a second, how do you know you're not gonna get AIDS from the HIV virus?"

Dr. John Tisdale: The short answer is we cut out the bits that cause infection in HIV and we really replace that with the gene that's misspelled in sickle cell disease so that it transfers that instead of the infectious part.

Dr. Jon LaPook: The stakes here are enormous.

Dr. Francis Collins: Yes.

Dr. Jon LaPook: There's really very little safety net here, right?

Dr. Francis Collins: Make no mistake, we're talking about very cutting-edge research where the certainty about all the outcomes is not entirely there. We can look back at the history of gene therapy and see there have been some tragedies.

Dr. Jon LaPook: Deaths?

Dr. Francis Collins: Yes.

In 1999, 18-year-old Jesse Gelsinger received altered DNA to treat a different genetic disease. He died four days later from a massive immune response. And in another trial, two children developed cancer.

Jennelle Stephenson understands. This is a trial with huge risks and no guarantees.

Jennelle Stephenson: This is it.

When she arrived at the NIH clinical center in December 2017, Jennelle asked her brother, Ray, for some help.

Jennelle Stephenson: There goes Ray cutting my hair. Oh, snip.

She decided to cut off all her hair, rather than watch it fall out from the massive dose of chemotherapy needed to suppress her immune system so her body wouldn't reject the altered stem cells.

Jennelle Stephenson: I don't know how to feel right now. I'm a little emotional. But I'm OK, it will grow back.

A few days after the chemotherapy, Jennelle received the infusion of genetically modified cells.

Dr. John Tisdale: Is it going good now?

Nurse: Yes.

Jennelle Stephenson: It's just a waiting game.

But the wait was a painful one. Not only for Jennelle, but also for her father Ray. Who did what little he could as the effects of the chemotherapy kicked in, stripping Jennelle's throat and stomach of their protective layers.

Jennelle Stephenson: Oh, that hurts.

She was unable to speak for a week and lost 15 pounds. And because having a severely weakened immune system means even a mild cold can turn deadly, Jennelle had to stay in the hospital for nearly a month.

Last spring, she moved back to Florida and returned to the NIH for periodic check-ups.

Dr. John Tisdale: These are her red blood cells.

It didn't take long for Dr. Tisdale to notice something was happening.

Dr. Jon LaPook: This is Jennelle before any treatment?

Dr. John Tisdale: Right. All across her blood you can see these really abnormal shapes. This one in particular is shaped like a sickle.

Nine months later, this is what Dr. Tisdale saw: not a sickle cell in sight.

Dr. Jon LaPook: Was there ever a moment where you saw one of these normal-looking smears and thought, "Is this the right patient?"

Dr. John Tisdale: Oh, absolutely. When you're a scientist, you're skeptical all the time. So, first thing you do is look and make sure it's that patient, go grab another one, make sure it's the same. And we've done all that. And, indeed, her blood looks normal.

Jiu-Jitsu Teacher: Move. Switch your arms and move.

Remember, Jennelle used to struggle just to walk up a flight of stairs...

Jiu-Jitsu Teacher: And you fall.

...and a fall like this would have landed her in the hospital.

Jiu-Jitsu Teacher: Boom. Yeah. Good job. You did it. Bam.

Dr. Jon LaPook: Jennelle. You look amazing.

Jennelle Stephenson: Thank you.

Dr. Jon LaPook: I have to say, I was a little nervous when you were thrown and you went down on the mat.

Jennelle Stephenson: It was nothing. It was nothing. My body just felt strong.

Dr. Jon LaPook: Tell me about the adjustment that you need to make to go from the old you to the new you.

Jennelle Stephenson: My body it almost felt like it was, like, itching to do more. And I was like, "All right, well, let's go swimming today." "Let's go to the gym today." I'm like, all right, my body loves this. I kinda like it because my, I guess all my endorphins started pumping.

Dr. Jon LaPook: The endorphin high, something you had never experienced.

Jennelle Stephenson: Never experienced before. Yup.

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Could gene therapy cure sickle cell anemia? - 60 Minutes ...

NLM ID: 101574143Index Copernicus Value 2016: 84.15

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Environmental pollution is "the tainting of the physical and organic segments of the earth/air framework to such a degree, that ordinary natural procedures are antagonistically influenced.

Pollution is the introduction of pollutants into the environment that can cause harm or uneasiness to mankind or other living creatures and can also adversely affect usefulness of a resources of earth. Pollutants can be synthetic substances, or energy, for example: noise, heat or light.

Different types os Environmental pollution are:Air pollution, Water pollution, Noise pollution, Light pollution, Soil pollution, Radioactive pollution, Thermal pollution, Plastic pollution etc.

Down syndrome is one of the most common genetic disorder that affects both physical and mental ability. It is caused by a gene problem before birth.Generally a normal person posses 46 chromosomes but a person with Down Syndrome has 47 chromosomes.There are three different types of Down syndrome: trisomy, translocation, and mosaicism. Symptoms include short head,short neck,poor muscle tone, excessive flexibility etc.

Down Syndrome results when each cell in the body possess three copies of chromosome 21 instead of two copies. Extra copies of genes on chromosome 21 results in the disruption of normal function and development of the body which increases the risk of health problems. Down Syndrome occurs when part of chromosome gets attached to another chromosome during the formation of reproductive cells or embryo. Affected people possess two normal copies of chromosome 21 and one extra chromosome that is attatched to other.

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Genetic mutation is a permanent change in the DNA.Mutations may or may not produce changes in the organism.Hereditary mutations and Somatic mutations are the two types of Gene mutations.Former type is inherited from the parents and are present in every cell of the human body whereas latter type may occur at some point of life time due to environmental factors.

Certain enzymes repair gene mutations that could cause a genetic disorder. These enzymes identify and repair mistakes in DNA before the gene is expressed and an altered protein is produced. When a mutation alters a protein, it can disrupt normal development. Mutation may occur from a single DNA to a large segment of chromosome that involves multiple genes.

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Sickel cell anemia is a blood disorder caused by an abnormality in haemoglobin molecule in red blood cells.Person inherited by Sickle-cell disease has two abnormal copies of haemoglobin gene.Normal red blood cells are round and flexible whereas sickled red blood cells appear in sickle-shape.Abnormal haemoglobin forms strands that change red blood cells to that form and hence they accumulate at the branches of the veins and blocks the flow of blood.As haemoglobin is responsible for carrying of oxygen throught out the body,there may be chronic attacks due to lack of oxygen supply.

Mutations in HBB gene results in Sickle Cell disease. Haemoglobin consists of four subunits.Two subunits are Alpha-globin and other two are Beta-globin. HBB gene is responsible for making instructions in the production of Beta-globin. Hence mutations in HBB gene results in different abnormal versions of beta-globin.These abnormal versions may distort red blood cells into sickle shape.

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It is a type of disease that causes progressive weakness and loss of muscle mass. Here the process of mutation get involved in the production of proteins that are required to build a healthy muscle.Some types of Muscular dystrophy are Myotonic, Facioscapulohumeral , Congenital, Limb-girdle. It occurs when one of the genes responsible for production of proteins is defective.But some of them occur in the early stage of embryo and is passed to the next generation.

Duchenne Muscular Dystrophy is the most common form and mostly affect boys. It is caused due to the absence of dystrophin,a protein involved in maintining the integrity of muscle. Facioscapulohumeral Muscular Dystrophy generally begins at the teenage age and causes progressive weakness in muscles of face, arms, legs, shoulders and chest. Myotonic Muscular Dystrophy is the most common form and causes cataracts, cardiac abnormalities and endocrine substances.

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Cystic fibrosis is a disorder caused by the presence of mutations in both the copies of the gene which is responsible for the protein cystic fibrosis transmembrane conductance regulator.It affects the cells that produce mucus, sweat and digestive juices.These fluids are thin and slippery but a defective gene causes these secretions to become thick ,thus blocking the passages in the lungs and pancreas.

Mutations in CFTR gene results in Cystic fibrosis. CFTR gene enables instructions for transportation of chloride ions into and out of the cells. Mutations in the CFTR gene disrupts the function of chloride channels that prevents the flow of chloride ions and water across cell membranes. As a result organs produce mucus that is thick and sticky which clogs the airways and ducts resulting isevere chronic attacks.

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An Auto immune disease develops when the immune system responsible for defending the body against diseases fights against the healthy cells. Here the immune system fails to differentiate healthy tissues and antigens, as a result the body sets off a reaction that destroy normal tissues.Some unknown trigger happens to confuse the immune system and instead of fighting against the infections it destroys the bodys own tissues.

Areas often affected by autoimmune disease include blood vessels, connective tissues, endocrine glands, joints, muscles, red blood cells, skin. Some common symptoms of autoimmune disease include fatigue, fever, joint pain, and rash. Some common autoimmune disorders include Addisons disease, Multiple Sclerosis, Type 1 diabetes, Sjogren syndrome, Reactive Arthritis, Dermatomyositis, Pernicious anemia, Celiac disease. This disorder may result in destruction of body tissue, abnormal growth of an organ, changes in organ function.

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Mitochiondrial disease is a group of disorder caused by dysfunctional mitochondria. Mytochondria are responsible for generation of 90% of energy required by the body to sustain life and growth.These are also known as the power house of the cell.They contain tiny packages of enzymes that converts nutrients into energy. This disease is caused by mutations in mitochondrial DNA and its failure in function may ultimately lead to cell death.

Symptoms include loss of motor control, muscle weakness and pain,swallowing difficulties,liver disease,diabetes,cardiac disease,gastro-intestinal disorders and developmental delay.Ecamples on mitochondrial diseases include dementia,Diabetes mellitus and deafness,Leigh syndrome,neuropathy,Myoclonic epilepsy,strke-like symptoms,mtDNA deletion.

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Congenial syndromes is a disease that exists before birth.These are characterized by structural deformities and defects are involved in developing fetus.Defects may be due to genetic or environmental factors.The outcome of the disorder may be because of mothers diet, vitamin intake,glucose levels prior to ovulation. Paternal exposures prior to conception and during pregnancy increases the risk of this disease.It is caused by multiple mutations of the fibroblast growth factor receptor 2 gene.

Defects may include errors of morphogenesis,infection,epigenetic modifications or a chromosomal abnormality.The causes of this syndrome may be due to Fetal alcohol exposure,Toxic substances,Paternal smoking,Infections,Lack of nutrients,Physical restraint,Genetic causes,Socioeconomic status,Role of radiation,Fathers age.

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Reye syndromes is a disease that causes swelling of the brainand liver .The actual cause is unknown but studies has shown that Aspirin is related to the cause of this disease generally in children and teenagers recovering from flu illness.The symptoms are vomiting, nausea, confusion,lethargy,coma, irritable and aggressive behavior.Abnormal laboratoty tests include rise in lever enzymes, ammonia levels and low serum glucose levels.

It is believed that tiny structures within the cell called the mitochondria become damaged. Mitochondria provide cells with energy to the liver for many of the vital functions such as filtering toxins from blood and regulating blood sugar levels. Failure of energy supply to the liver may result in build up of toxic chemicals in the blood which can damage the entire body.It is often seen in children ages 4 to 12. Symptoms are so mild that they go unnoticed. Early detection and treatment are critical but the chances for a successful recovery are greater when Reye Syndrome is treated at its earliest stages. Complications may include coma, permanent brain damage, seizures.

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Patau syndromes is a disorder caused by chromosomal abnormality.It occurs when some or all the cells contain extra copy of the chromosome 13.This restricts the normal functioning ,growth and development of the organs resulting in intellectual disability and physical abnormalities. It is also called Trisomy 13.It also can occur when part of chromosome gets attatched to another chromosome during the formation of embryo.

Most cases of trisomy 13 are not inherited and results from the random events during the formation of eggs and sperm. An error in cell division may result in abnormal number of chromosome. If this extra copy contributes in the genetic makeup of child then the child possess an extra chromosome 13 in each cell of the body resulting in the physical abnormalities in most of the parts.

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Fragile syndrome is a genetic disorder that results in intellectual disability.Mutations in the FMRI gene causes this disease. This gene is responsible for the preparation of a protein ,FMRP.This protein regulates the production of other proteins and is necessary for the development of synapses which are the connections between nerve cells.Mutations in FMRI prevents the production of FMRP ,thus disturbing the nervous system.

Males are severely affected by this disorder than females.Affected individuals usually have delayed development of speech and language by age 2.Children with fragile X syndrome may also have anxietyand hyperactive behavior such as impulsive actions. Fragile X syndrome is inherited in an X-linked dominant pattern. This condition is considered as X-linked since the mutated gene that causes the disorder is located on X chromosome.

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Angelman syndrome is a genetic disorder that affects the nervous system.Characteristic features include happy demeanor,intelluctual disability,speech impairment,walking and balancing disorders.This arises when segment of the maternal chromosome 15 containing the gene UBE3 A is deleted or undergoes mutation.People inherit one copy of this gene from each parent and both the copies remain active in many of the body tissues.But due to genetic mutations, gene may become active or get deleted in some parts of the brain resulting in intellectual disability.

Angelman Syndrome may also be caused by a chromosomal rearrangement called a translocation or by a mutation or other defect in the region of DNA that controls the activation of UBE3A gene. In some people with angelman syndrome the loss of a gene called OCA2 is associated with light colored hair and fair skin. This gene is located on the segment of chromosome 15 that is deleted in people with this disorder. Most cases of this syndrome are not inherited.

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Tay-Sachs is a genetic disorder that destroys the nerve cells in the brain and spinal cord. Characteristic features include weakening of muscles,intellectual disability,vision and hearing loss,paralyses.Mutations in the HEXA gene causes this disease .This gene is responsible for the production of an enzyme in lysosome which plays a critical role in the brain and spinal cord.This enzyme breaks down the toxic substances in the cell.Mutations in the HEXA gene causes failure in the production of enzyme resulting in the accumulation of toxic substances in the cells leading to damage in the neurons of the brain and spinal cord.

Since Tay-Sachs disease impairs the function of a lysosomal enzyme this condition is sometimes referred to as a lysosomal storage disorder.This condition is inherited in which both the copies if the gene undergoes mutations. Persons with Tay- Sachs disease experience vision and hearing loss, intellectual disability and paralysis. An eye abnormality called a cherry-red spot is the characteristic feature of this disorder.

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Prenatal genetic testing is meant to evaluate the chance of exhibiting genetic disorders in their unborn children.The tests are usually done between 10th and 13th week of pregnancy . These tests involves the measurement of certain levels of substances in the mothers blood and obtaining an ultrasound.These tests are meant to evaluate the genetic material of the fetus for any genetic disorders.It is also useful to diagnose high risk pregnancies.

Genetic tests are performed on a sample of blood,hair,skin,amniotic fluid or other tissue.A positive test result means that the laboratory found a change in a particular gene, chromosome or a protein. A negative test result means that the laboratory did not find a change in the gene, chromosome or a protein that is under consideration.

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Carcinogenesis, Genetic Engineering,Obstetrics & Gynecology, Genetic Testing, Fetal Diagnosis and Therapy, Clinical Genetics, Prenatal Diagnosis, Journal of Midwifery & Womens Health, Prenatal genetic testing Journals,Genetic Testing Articles,Genetic Journals

Genes hold DNA that are responsible for giving instructions in the production of proteins.Mutations in genes may cause failure in the working of proteins leading to a condition called genetic disorder.These disorders may be inherited form parents or may occur at any point of lifetime.Genetic disorder may result in the addition or reduction in the number of chromosomes.

The four groups of genetic disorders are Single gene disorders, chromosome abnormalities, mitochondrial disorders, and multifactorial disorders. The four main ways of inheriting an altered gene are autosomal dominant, autosomal recessive, X-linked dominant and X-linked recessive. Genetic disorders may or may not be heritable. In non-heritable genetic disorders defects may be due to mutations in the DNA.

Related Journals of Genetic Disorders

Genetic Engineering, Stem Cell, Journal of Genetic Disorders & Genetic Reports, Journal of Medical Genetics, Journal of Genetic Mutation Disorders, Source Journal of Genetic Disorders, Genetic Disorders, Genes and Diseases, Genetic disorders Journals,Genetic Disorder Articles

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Journal of Genetic Syndromes & Gene Therapy

Gene therapy is the addition of new genes to a patient's cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because thats what viruses evolved to do with their own genetic material.

The treatment, which was first tested in humans in 1990, can be performed inside or outside of the body. When its done inside the body, doctors may inject the virus carrying the gene in question directly into the part of the body that has defective cells. This is useful when only certain populations of cells need to be fixed. For example, researchers are using it to try to treat Parkinson's disease, because only part of the brain must be targeted. This approach is also being used to treat eye diseases and hemophilia, an inherited disease that leads to a high risk for excess bleeding, even from minor cuts.

Early in-the-body gene therapies used a virus called adenovirusthe virus behind the common coldbut the agent can cause an immune response from the body, putting a patient at risk of further illness. Today, researchers use a virus called adeno-associated virus, which is not known to cause any disease in humans. In nature, this agent needs to hitch a ride with an adenovirus, because it lacks the genes required to spread itself on its own. To produce an adeno-associated virus that can carry a therapeutic gene and live on its own, researchers add innocuous DNA from adenovirus during preparation.

In-the-body gene therapies often take advantage of the natural tendency of viruses to infect certain organs. Adeno-associated virus, for example, goes straight for the liver when it is injected into the bloodstream. Because blood-clotting factors can be added to the blood in the liver, this virus is used in gene therapies to treat hemophilia.

In out-of-the-body gene therapy, researchers take blood or bone marrow from a patient and separate out immature cells. They then add a gene to those cells and inject them into the bloodstream of the patient; the cells travel to the bone marrow, mature and multiply rapidly, eventually replacing all of the defective cells. Doctors are working on the ability to do out-of-the-body gene therapy to replace all of a patient's bone marrow or the entire blood system, as would be useful in sickle-cell anemiain which red blood cells are shaped like crescents, causing them to block the flow of blood.

Out-of-the-body gene therapy has already been used to treat severe combined immunodeficiencyalso referred to as SCID or boy-in-the-bubble syndromewhere patients are unable to fight infection and die in childhood. In this type of gene therapy, scientists use retroviruses, of which HIV is an example. These agents are extremely good at inserting their genes into the DNA of host cells. More than 30 patients have been treated for SCID, and more than 90 percent of those children have been cured of their disorderan improvement over the 50 percent chance of recovery offered by bone marrow transplants.

A risk involved with retroviruses is that they may stitch their gene anywhere into DNA, disrupting other genes and causing leukemia. Unfortunately, five of the 30 children treated for SCID have experienced this complication; four of those five, however, have beaten the cancer. Researchers are now designing delivery systems that will carry a much lower risk of causing this condition.

Although there are currently no gene therapy products on the market in the U.S., recent studies in both Parkinson's disease and Leber congenital amaurosis, a rare form of blindness, have returned very promising results. If these results are borne out, there could be literally hundreds of diseases treated with this approach.

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How does gene therapy work? - Scientific American

Lexis Conferencesareproudly announcedthe conference The Gene Therapy and Epigenetics 2019 which is going to take place in London, UK during September 9-10, 2019. Lexis invites the conventioneer from around the globe to attend The Gene Therapy and Epigenetics 2019 with the Theme: Novel Approaches in Human Genome and Genetic Disorders.

Gene Therapy and Epigenetics Conferences will incorporate incite Keynote presentations/Plenary talks, Workshops, Symposiums, Special sessions, Poster presentations, Video sessions, and Exhibitions. This trending topic needs an exchange of ideas, discussions, and debates to reach the new dimension in the topic. The Gene Therapy and Epigenetics 2019 is a platform to showcase your abilities to the competitive world.

ABOUT GENE THERAPY AND EPIGENETICS CONFERENCE 2019:

Gene therapyisa unique technique used in medical treatment that uses specific types of genes to treat several types of diseases. Gene therapy advancement is meant to cure rare diseases and even some inherited diseases, which are caused by a mutated or faulty gene.Gene Therapyis also used to treat several Genetics disorders, wherein the mutated defective gene is replaced with the functional gene.

Gene therapyis the one which most vast topics carried out by researchers all over the world for the prevent or treat of several diseases such as immune deficiencies, hemophilia, Parkinsons disease, Cancer, and even HIV, through different approaches. Three primary approaches that are being studied and practiced in thegene therapyare replacement of the mutated disease-causing gene with the healthy gene, inactivation of the mutated gene, and introduction of the new gene to fight against the disease. In the gene therapy treatment, a functional gene is inserted into the genome of an individuals cells and tissues by using a carrier known as vector. Viruses are the most common type of vectors used ingene therapy, which is genetically altered to carry the normal human DNA.

Over the last few years,gene therapyhas emerged as a promising treatment option for several diseases including inherited disorders and certain types of cancers and viral infections. Scientists use these techniques to readily manipulate viral genomes, isolate genes and identify mutations involved in human disease, characterize and regulate gene expressions, and engineer various viral and non-viral vectors. Various long-term treatments for anemia, hemophilia, cystic fibrosis, muscular dystrophy, Gaucher's disease, lysosomal storage diseases, cardiovascular diseases, diabetes and diseases of bones and joints are resolved through successful gene therapy and are elusive today.

Epigenetics is an extension of genetics and developmental biology, which involves the study of cellular and physiological trait variations initiated by external or environmental stimuli. Epigenetics deals with changes in gene expression caused by certain base pairs in DNA & RNA, which are turned off or turned on, through chemical reactions contrary to being affected by changes in the nucleotide sequence. Epigenetic alterations result into a change in phenotype, with the genotype of the organism being constant. Epigenetics changes are influenced by different factors, such as age, surrounding environment, lifestyle, disease state, and others.

Epigenetics can possibly be a key component in a worldview change of our comprehension of health and disease and generally change public health policies. Epigenetic modifications are ordinarily utilized amid the advancement and support of various cell composes, however defective epigenetic control can cause enduring harm, prompting tumor and different illnesses ranging from metabolic scatters, for example, diabetes to coronary illness and psychological well-being conditions.

DNA methylation and histone modification, for instance, are epigenetic forms wherein the alteration in gene expression is observed without the adjustment in the DNA Sequence. Ascend in tumor pervasiveness; enhanced financing and helps for R&D activities, a flood in an association between scholarly, pharmaceutical, and biotechnology organizations, and expanded utilization of epigenetics in non-Oncology infections are the key factors that impel the development of this market.

Epigenetics is the study of heritable changes in gene expression that do not involve changes to the underlying DNA Sequence. Which in turn affects how cells read the genes. Epigenetic modifications can manifest as commonly as the manner in which cells terminally differentiate to end up as skin cells, liver cells, brain cells, etc. Or, epigenetic change can have more damaging effects that can result in diseases like cancer. At least three systems including DNA methylation, histone modification and non-coding RNA (ncRNA)-associated gene silencing are currently considered to initiate and sustain epigenetic change. New and ongoing research is continuously uncovering the role of epigenetics in a variety of human disorders and fatal diseases.

WHO TO ATTENDGENE THERAPY AND EPIGENETICSEVENT:

DETAILS OF EPIGENETICS CONFERENCE 2019 IN LONDON, UK:

Lexisis organizing Gene Therapy and Epigenetics Conferencein 2019 in London. We organize Genetics and Molecular Biology Meetingslike Human Genetics, Stem Cell research, Cell and Gene Therapies, Epigenetics, Proteomics and in Biologylike Structural, Molecular, Cell, Plant and Animal.

IMPORTANCE AND SCOPE OF THE GENE THERAPY AND EPIGENETICS EVENT:

The global Gene Therapy market size was esteemed at USD 7.6 million of every 2017. It is evaluated to grow at a CAGR of more than 19.0% during the forecast period. Gene therapy marketsize is relied upon to achieve USD 39.54 million by 2026. Rising rivalry among makers and a high number of atoms in the pipeline is supporting the growth of the market.

Gene Therapy development is planned to cure rare diseases and even some inherited diseases, which are caused by a mutated or faulty gene. In addition, the consistently expanding requirement for new solutions for vagrant ailments and the rising incidence of cancer caused because of transformations in genes are probably going to mix up the interest for gene therapy.

As of early 2016, there was an excess of 1000 molecules in the pipeline in various clinical phases. However, around 76.0% of the atoms are in the formative or preclinical stages and anticipated that would hit the market in the late 2020's.

The global Epigenetics showcase was esteemed at US$ 4.63 Billion in 2017 and is expected to achieve US$ 16.50 Billion by 2026, growing at a CAGR of 15.03 % from 2018 to 2026.

North America(US and Canada) is the present pioneer in the worldwide epigenetics market and anticipated that would demonstrate predominance over the forecast period. Higher acknowledgment of more up to date advancements enormous interest in R&D and developed social insurance framework are the key factors contributing to the strength of this region.

On the other hand, Asia Pacific is anticipated to demonstrate the fastest market growth over the conjecture time frame fundamentally because of expanding human services spending and creating medicinal services framework. Noteworthy CRO activities in hubs, for example, Indiaalso feature the rapid pace of Asia Pacific market.

The epigenetics market is fragmented in view of product, application, end user, and topography. In view of the item, it is separated into proteins, kits & assays, instruments, and reagents.

Based on the end user, the market is arranged into academic & research institutes, pharmaceutical organizations, biotechnology companies & contract research organizations (CROs). Geographically, the market is analyzed across North America, Europe (Germany, UK, France, and Rest of Europe), Asia-Pacific (Japan, China, India, and rest of the APAC), and LAMEA.

Excerpt from:
Genetics Conferences 2019: Gene Therapy, Cell Therapy ...

Gene therapy, the use of genetic material to treat a disease or disorder, is making strides in muscular dystrophy. Although the approach is still considered experimental, studies in animal models have shown promising results and clinical trials in humans are underway.

Gene therapy has the potential to help people with inherited disorders, in which a gene mutation causes cells to produce a defective protein or no protein at all, leading to disease symptoms.

To deliver the genetic material to the cells, scientists use a tool called a vector. This is typically a virus that has been modified so that it doesnt cause disease. It is hoped that the vector will carry the therapeutic gene into the cells nucleus, where it will provide the instructions necessary to make the desired protein.

The most common form of muscular dystrophy, Duchenne muscular dystrophy, is caused by a mutation in the DMD gene, which codes for a protein called dystrophin. Dystrophin is part of a protein complex that strengthens and protects muscle fibers. When the cells dont have functional dystrophin due to the gene mutation, muscles progressively weaken. Scientists think that supplying a gene that codes for a functional form of dystrophin might be an effective treatment for Duchenne muscular dystrophy.

Using gene therapy to deliver a correct form of the dystrophin gene has been challenging because of the size of the DMD gene, which is the largest gene in the human genome so it does not fit into commonly used vectors.

Scientists are having more success with a shortened version of the DMD gene that produces a protein called micro-dystrophin. Even though its a smaller version of dystrophin, micro-dystrophin includes key elements of the protein and is functional.

Administering a gene for micro-dystrophin to golden retriever dogs that naturally develop muscular dystrophy showed promising results in a study published in July 2017. Muscular dystrophy symptoms were reduced for more than two years following the treatment and the dogs muscle strength improved. The gene was delivered using a recombinant adeno-associated virus, or rAAV, as the vector.

A similar therapy is now being tested in people in a Phase 1/2 clinical trial (NCT03375164)at Nationwide Childrens Hospital in Columbus, Ohio. A single dose of the gene therapytreatment containing the gene encoding for micro-dystrophinwill be infused into the blood system of 12 patients in two age groups: 3 months to 3 years, and 4 to 7 years. The first patient in the trial, which is recruiting participants, already has received the treatment, according to a January 2018 press release.

The biopharmaceutical company Sarepta Therapeutics is contributing funding and other support to the project.

Sarepta is developing another potential gene therapy for Duchenne muscular dystrophy where rather than targeting the DMD gene that codes for dystrophin, the therapy will be used to try to increase the expression of a gene called GALGT2. The overproduction of this gene is thought to produce changes in muscle cell proteins that strengthen them and protect them from damage, even in the absence of functional dystrophin.

A Phase 1/2a clinical trial (NCT03333590) was launched in November 2017 at Nationwide Childrens Hospital for the therapy, called rAAVrh74.MCK.GALGT2.

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Muscular Dystrophy Newsis strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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Gene Therapy in Muscular Dystrophy

Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. The U.S. Food and Drug Administrationapprovedthe first gene therapy for an inherited disease a genetic form of blindness in December 2017.

Sickle cell anemia is caused by a mutation in the HBB gene which provides the instructions to make part of hemoglobin, the protein in red blood cells that carries oxygen.

Researchers are working on two different strategies to treat sickle cell anemia with gene therapy. Both of these strategies involve genetically altering the patients own hematopoietic stem cells. These are cells in the bone marrow that divide and specialize to produce different types of blood cells, including the red blood cells.

One strategy is to remove some of the patients hematopoietic stem cells, replace the mutated HBB gene in these cells with a healthy copy of the gene, and then transplant those cells back into the patient. The healthy copy of the gene is delivered to the cells using a modified, harmless virus. These genetically corrected cells will then hopefully repopulate the bone marrow and produce healthy, rather than sickled, red blood cells.

The other strategy is to genetically alter another gene in the patients hematopoietic stem cells so they boost production of fetal hemoglobin a form of hemoglobin produced by babies from about seven months before birth to about six months after birth. This type of hemoglobin represses sickling of cells in patients with sickle cell anemia, but most people only produce a tiny amount of it after infancy. Researchers aim to increase production of fetal hemoglobin in stem cells by using a highly specific enzyme to cut the cells DNA in the section containing one of the genes that suppress production of fetal hemoglobin. When the cell repairs its DNA, the gene no longer works and more fetal hemoglobin is produced.

Gene therapy offers an advantage over bone marrow transplant, in that complications associated with a bone marrow donation now the only cure for the disease such as finding the right match are not a concern.

Twelve clinical trials studying gene therapy to treat sickle cell anemia are now ongoing. Nine of the 12 are currently recruiting participants.

Four trials (NCT02186418, NCT03282656, NCT02247843, NCT02140554) are testing the efficacy and safety of gene therapy to replace the mutated HBB gene with a healthy HBB gene. These Phase 2 trials are recruiting both children and adults in the United States and Jamaica.

Three trials (NCT02193191, NCT02989701, NCT03226691) are investigating the use ofMozobil (plerixafor) in patients with sickle cell anemia to increase the production of stem cells to be used for gene therapy. This medication is already approved to treat certain types of cancer. All three are recruiting U.S. participants.

One trial (NCT00669305) is recruiting sickle cell anemia patients in Tennessee to donate bone marrow to be used in laboratory research to develop gene therapy techniques.

The final study(NCT00012545) is examining the best way to collect, process and store umbilical cord blood from babies with and without sickle cell anemia. Cord blood contains abundant stem cells that could be used in developing gene therapy for sickle cell anemia. This trial is open to pregnant women in Maryland both those who risk having an infant with sickle cell anemia, and those who do not.

One clinical trial (NCT02151526) conducted in France is still active but no longer recruiting participants. It is investigating the efficacy of gene therapy in seven patients. For the trial, a gene producing a therapeutic hemoglobin that functions similarly to fetal hemoglobin is introduced into the patients stem cells. A case studyfrom one of the seven was published in March 2017; it showed that the approach was safe and could be an effective treatment option for sickle cell anemia.

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Sickle Cell Anemia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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Gene Therapy - Sickle Cell Anemia News

NEWARK,March 15, 2018 The New Jersey Innovation Institute, (NJII) an NJIT Corporation, has announced that its Cell and Gene Therapy Development Center has launched a training program to upgrade the knowledge and skills of biopharmaceutical professionals in the processing of new, breakthrough classes of biologic therapies.

The workforce training program is in response to increasing demands from the biopharmaceutical industry for engineers and scientists to be trained in manufacturing and processing of the newest biologic and immunotherapies such as advanced CAR-T cancer therapy. The program will combine lectures and hands-on training to introduce the newest approaches and technologies applied to the development and production of these innovative therapies.

NJII President and CEO, Dr. Donald H. Sebastian said, The pharmaceutical industry faces formidable challenges as it adapts to the new culture of biotechnology. This training initiative demonstrates NJIIs commitment to advance cell and gene therapy manufacturing and processing innovation.

Dr. Haro Hartounian, NJIIs executive director, biotechnology and pharmaceutical innovation stated, The pace of development in cell and gene therapy is unprecedented in the biopharmaceutical industry. It is imperative that engineers and scientists are proficient not only in in the latest processing techniques, but that they also acquire a basic understanding of the underlying protocols. Our instructional team composed of industry and university faculty experts is ideally structured to meet the needs of the industry for training of their workforce in the manufacturing and processing of these novel biopharmaceuticals.

The New Jersey Innovation Institute (NJII) is an NJIT corporation that applies the intellectual and technological resources of the states science and technology university to challenges identified by industry partners. Through its Innovation Labs (iLabs), NJII brings NJIT expertise to key economic sectors, including healthcare delivery systems, bio-pharmaceutical production, civil infrastructure,defense and homeland security, and financial services.

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New Jersey Innovation Institutes Cell & Gene Therapy ...

Scientists have promised that gene therapy will be the next big leap for medicine. It's a term that's tossed about regularly, but what is it exactly? Trace shows us how scientists can change your very genetic code.

Read More:

How does gene therapy work?http://ghr.nlm.nih.gov/handbook/thera..."Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein."

Gene therapy trial 'cures children'http://www.bbc.co.uk/news/health-2326..."A disease which robs children of the ability to walk and talk has been cured by pioneering gene therapy to correct errors in their DNA, say doctors."

Gene therapy cures diabetic dogshttp://www.newscientist.com/article/d..."Five diabetic beagles no longer needed insulin injections after being given two extra genes, with two of them still alive more than four years later."

Gene Therapy for Cancer: Questions and Answershttp://www.cancer.gov/cancertopics/fa..."Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight or prevent disease."

How does gene therapy work?http://www.scientificamerican.com/art..."Gene therapy is the addition of new genes to a patient's cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because that's what viruses evolved to do with their own genetic material."

Gene therapy cures leukaemia in eight dayshttp://www.newscientist.com/article/m...eight-days.htmlWITHIN just eight days of starting a novel gene therapy, David Aponte's "incurable" leukaemia had vanished. For four other patients, the same happened within eight weeks, although one later died from a blood clot unrelated to the treatment, and another after relapsing.

Cell Therapy Shows Promise for Acute Type of Leukemiahttp://www.nytimes.com/2013/03/21/hea..."A treatment that genetically alters a patient's own immune cells to fight cancer has, for the first time, produced remissions in adults with an acute leukemia that is usually lethal, researchers are reporting."

Watch More:Tricking the Immune Systemhttp://www.youtube.com/watch?v=Kr_HRl...Babies with 3 Parents?!http://www.youtube.com/watch?v=jQxsW_...Pick Your Poison: Cyanidehttp://www.youtube.com/watch?v=JDBrdE...____________________

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How Does Gene Therapy Work?

Gene therapy is a cancer treatment that is still in the early stages of research.

Genes are coded messages that tell cells how to make proteins. Proteins are the molecules that control the way cells behave. Our genes decide what we look like and how our body works.We have many thousands of separate genes.

Genes are made ofDNAand they are in the nucleus of the cell. The nucleus is the cell's control centre.Genes are grouped together to make chromosomes. We inherit half our chromosomes from our mother and half from our father.

Cancer cells are different from normal cells. They have changes (called faults or mutations) in several of their genes which make them divide too often and form a tumour. The genes that are damaged mightbe:

Many gene changes thatmake a cell become cancerous are caused by environmental or lifestyle factors. A small numberof people haveinherited faulty genes that increase their risk of particular types of cancer.

Gene therapy is a type of treatment which uses genes to treat illnesses. Researchers have been developing differenttypes of gene therapyto treat cancer.

The ideas for these new treatments have come about because we are beginning to understand how cancer cells are different from normal cells. It is stillearly days for this type of treatment. Some of these treatments are being looked at in clinical trials. Otherscan now be used for some people with types of cancer such as melanoma skin cancer.

Getting genes into cancer cells is one of the most difficult aspects of gene therapy. Researchers are working on finding new and better ways of doing this. The gene is usually taken into the cancer cell by a carrier called a vector.

The most common types of carrier used in gene therapy are viruses because they can enter cells and deliver genetic material. The viruses have been changed so that they cannot cause serious disease but they may still cause mild, flu-like symptoms.

Some viruses have been changed in the laboratory so that they target cancer cells and not healthy cells. So they only carry the gene into cancer cells.

Researchers are testing other types of carrier such as inactivated bacteria.

Researchers are looking at different ways of using gene therapy:

Some types of gene therapy aim to boost the body's natural ability to attack cancer cells. Ourimmune systemhas cells that recognise and kill harmful things that can cause disease, such as cancer cells.

There are many different types of immune cell. Some of them produce proteins that encourage other immune cells to destroy cancer cells. Some types of therapy add genes to a patient's immune cells. Thismakes them better at finding or destroying particular types of cancer.

There are a few trials using this type of gene therapy in the UK.

Some gene therapies put genes into cancer cells to make the cells more sensitive to particular treatments. The aim is to make treatments,such as chemotherapy or radiotherapy, work better.

Some types of gene therapy deliver genes into the cancer cells that allow the cells to change drugs from an inactive form to an active form. The inactive form of the drug is called a pro drug.

First of all you have treatment with thecarrier containing the gene, then you havethe pro drug.The pro drug circulates in the body and doesn't harm normal cells. But when it reaches the cancer cells, it is activated by the gene and the drug kills the cancer cells.

Some gene therapies block processes that cancer cells use to survive. For example, most cells in the body are programmed to die if their DNA is damaged beyond repair. This is called programmed cell death or apoptosis. Cancer cells block this process so they don't die even when they are supposed to.

Some gene therapy strategies aim to reverse this blockage. Researchers are looking at whetherthese new types of treatment will make the cancer cells die.

Some viruses infect and kill cells. Researchers are working on ways to change these viruses so they only target and kill cancer cells, leaving healthy cells alone.

This sort of treatment uses the viruses to kill cancer cells directly rather than to deliver genes. So it is not cancer gene therapy in the true sense of the word. But doctors sometimes refer to it as gene therapy.

An example is a drug called T-VEC (talimogene laherparepvec), also known as Imlygic. It uses a strain of the cold sore virus (herpes simplex virus) that has been changed by altering the genes that tell the virus how to behave. It tells the virus to destroy the cancer cells and ignore the healthy cells.

T-VEC is now available as a treatment for melanoma skin cancer. It can be used to treat some people with melanomawhose cancer cannot be removed with surgery. It is also being looked at in trials for head and neck cancer. You have T-VEC as an injection directly into the melanoma or head and neck cancer.

Use the tabs along the top to look at recruiting, closed and results.

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Gene therapy | Cancer in general | Cancer Research UK

In this impressive, meticulously researched study of the exciting new developments in gene therapy, geneticist and journalist Lewis (Human Genetics) looks closely at the history of setbacks plaguing the treatment of rare genetic diseases as well as recent breakthroughs...Yet with each success, as Lewis recounts in this rigorous, energetic work, possibilities in treating HIV infection and dozens of other diseases might be around the next corner. Publisher's Weekly (starred review)

A fascinating account of groundbreaking science and the people who make it possible. Kirkus

Ricki Lewis gives us the inspiring story of gene therapy as told through Corey's eyes--literally. Her book delves into the challenges modern medicine faces--both in its bitter disappointments and great successes--but it goes much deeper than that. With empathy and grace, Lewis shows us the unimaginable strength of parents with sick children and the untiring devotion of the physicians who work to find the forever fix' to save them. But best of all Lewis gives us a story of profound hope. Molly Caldwell Crosby, author of The American Plague: The Untold Story of Yellow Fever, the Epidemic that Shaped Our History and Asleep: The Forgotten Epidemic that Remains One of Medicine's Greatest Mysteries

The Forever Fix is a wonderful story told by one of our most gifted science and medical writers. In the tradition of Siddhartha Mukherjee's The Emperor of All Maladies, Ricki Lewis explains complex biological processes in extremely understandable ways, ultimately providing crucial insights into the modeling of disease and illustrating how gene therapy can treat and even potentially cure the most challenging of our health conditions. Dennis A. Steindler, Ph.D., former Executive Director of the McKnight Brain Institute, University of Florida

Ricki Lewis has written a remarkable book that vividly captures the breathtaking highs and devastating lows of gene therapy over the past decade while giving ample voice to all sides -- the brave patient volunteers, their parents and physicians. The Forever Fix is required reading as we dare to dream of curing a host of genetic diseases. Kevin Davies, Founding editor of Nature Genetics; author of The $1,000 Genome and Cracking the Genome

In 'The Forever Fix,' Ms. Lewis chronicles gene therapy's climb toward the Peak of Inflated Expectations over the course of the 1990s. A geneticist and the author of a widely used textbook, she demonstrates a mastery of the history. The Wall Street Journal

An engaging and accessible look at gene therapy. Times Union

Medical writer Ricki Lewis interweaves science, the history of medical trial and error, and human stories from the death in 1999 of teenager Jesse Gelsinger, from a reaction to gene therapy intended to combat his liver disease, to radical successes in some children with adenosine deaminase deficiency. Nature

Lewis adeptly traverses the highs and lows of gene therapy and explores its past, present, and future through the tales of those who've tested its validity. The Scientist

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The Forever Fix: Gene Therapy and the Boy Who Saved It ...

Gene therapy is a technology aimed at correcting or fixing a gene that may be defective. This exciting and potentially transformative area of research is focused on the development of potential treatments for monogenic diseases, or diseases that are caused by a defect in one gene.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patients cells to compensate for a defective one, using a viral vector.

The technology involves the introduction of genetic material (DNA or RNA) into the body, often through delivering a corrected copy of a gene to a patients cells to compensate for a defective one, using a viral vector.

Viral vectors can be developed using adeno-associated virus (AAV), a naturally occurring virus which has been adapted for gene therapy use. Its ability to deliver genetic material to a wide range of tissues makes AAV vectors useful for transferring therapeutic genes into target cells. Gene therapy research holds tremendous promise in leading to the possible development of highly-specialized, potentially one-time delivery treatments for patients suffering from rare, monogenic diseases.

Pfizer aims to build an industry-leading gene therapy platform with a strategy focused on establishing a transformational portfolio through in-house capabilities, and enhancing those capabilities through strategic collaborations, as well as potential licensing and M&A activities.

We're working to access the most effective vector designs available to build a robust clinical stage portfolio, and employing a scalable manufacturing approach, proprietary cell lines and sophisticated analytics to support clinical development.

In addition, we're collaborating with some of the foremost experts in this field, through collaborations with Spark Therapeutics, Inc., on a potentially transformative gene therapy treatment for hemophilia B, which received Breakthrough Therapy designation from the US Food and Drug Administration, and 4D Molecular Therapeutics to discover and develop targeted next-generation AAV vectors for cardiac disease.

Gene therapy holds the promise of bringing true disease modification for patients suffering from devastating diseases, a promise were working to seeing become a reality in the years to come.

Excerpt from:
Gene Therapy | Pfizer: One of the world's premier ...

Overview

Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease.

Genes contain your DNA the code that controls much of your body's form and function, from making you grow taller to regulating your body systems. Genes that don't work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.

Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease.

Researchers are investigating several ways to do this, including:

Gene therapy has some potential risks. A gene can't easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector.

The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells' genes. Researchers remove the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease.

This technique presents the following risks:

The gene therapy clinical trials underway in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research.

Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body.

Your specific procedure will depend on the disease you have and the type of gene therapy being used.

For example, in one type of gene therapy:

Viruses aren't the only vectors that can be used to carry altered genes into your body's cells. Other vectors being studied in clinical trials include:

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:

But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:

Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Dec. 29, 2017

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Gene therapy - Mayo Clinic

A panel of experts has recommended that the Food and Drug Administration approve a treatment developed by Spark Therapeutics for a rare form of blindness. Spark Therapeutics hide caption

A panel of experts has recommended that the Food and Drug Administration approve a treatment developed by Spark Therapeutics for a rare form of blindness.

Gene therapy, which has had a roller-coaster history of high hopes and devastating disappointments, took an important step forward Thursday.

A Food and Drug Administration advisory committee endorsed the first gene therapy for an inherited disorder a rare condition that causes a progressive form of blindness that usually starts in childhood.

The recommendation came in a unanimous 16-0 vote after a daylong hearing that included emotional testimonials by doctors, parents of children blinded by the disease and from children and young adults helped by the treatment.

"Before surgery, my vision was dark. It was like sunglasses over my eyes while looking through a little tunnel," 18-year-old Misty Lovelace of Kentucky told the committee. "I can honestly say my biggest dream came true when I got my sight. I would never give it up for anything. It was truly a miracle."

Several young people described being able to ride bicycles, play baseball, see their parents' faces, read, write and venture out of their homes alone at night for the first time.

"I've been able to see things that I've never seen before, like stars, fireworks, and even the moon," Christian Guardino, 17, of Long Island, N.Y., told the committee. "I will forever be grateful for receiving gene therapy."

The FDA isn't obligated to follow the recommendations of its advisory committees, but it usually does.

If the treatment is approved, one concern is cost. Some analysts have speculated it could cost hundreds of thousands of dollars to treat each eye, meaning the cost for each patient could approach $1 million.

Spark Therapeutics of Philadelphia, which developed the treatment, hasn't said how much the company would charge. But the company has said it would help patients get access to the treatment.

Despite the likely steep price tag, the panel's endorsement was welcomed by scientists working in the field.

"It's one of the most exciting things for our field in recent memory," says Paul Yang, an assistant professor of ophthalmology at the Oregon Health and Science University who wasn't involved in developing or testing the treatment.

"This would be the first approved treatment of any sort for this condition and the first approved gene therapy treatment for the eye, in general," Yang says. "So, on multiple fronts, it's a first and ushers in a new era of gene therapy."

Ever since scientists began to unravel the genetic causes of diseases, doctors have dreamed of treating them by fixing defective genes or giving patients new, healthy genes. But those hopes dimmed when early attempts failed and sometimes even resulted in the deaths of volunteers in early studies.

But the field may have finally reached a turning point. The FDA recently approved the first so-called gene therapy product, which uses genetically modified cells from the immune system to treat a form of leukemia. And last week, scientists reported using gene therapy to successfully treat patients suffering from cerebral adrenoleukodystrophy, or ALD, a rare, fatal brain disease portrayed in the film Lorenzo's Oil. Researchers are also testing gene therapy for other causes of blindness and blood disorders such as sickle cell disease.

The gene therapy endorsed by the committee Thursday was developed for RPE65-mutation associated retinal dystrophy, which is caused by a defective gene that damages cells in the retina. About 6,000 people have the disease worldwide, including 1,000 to 2,000 people in the United States.

The treatment, which is called voretigene neparvovec, involves a genetically modified version of a harmless virus. The virus is modified to carry a healthy version of the gene into the retina. Doctors inject billions of modified viruses into both of a patient's eyes.

In a study involving 29 patients, ages 4 to 44, the treatment appeared to be safe and effective. More than 90 percent of the treated patients showed at least some improvement in their vision when tested in a specially designed obstacle course. The improvement often began within days of the treatment.

"Many went from being legally blind to not being legally blind," said Albert Maguire, a professor of ophthalmology who led the study at the University of Pennsylvania, in an interview before the hearing.

The improvement varied from patient to patient, and none of the patients regained normal vision. But some had a significant increase in their ability to see, especially at night or in dim light, which is a major problem for patients with this condition.

"What I saw in the clinic was remarkable," Maguire told the committee. "Most patients became sure of themselves and pushed aside their guides. Rarely did I see a cane after treatment."

That was the case of Allison Corona, who's now 25 and lives in Glen Head, N.Y. She underwent the treatment five years ago as part of the study.

"My light perception has improved tremendously," Corona said during an interview before the hearing. "It's been life-changing. I am able to see so much better. I am so much more independent than what I was. It is so much better."

The patients have been followed for more than three years, and the effects appear to be lasting. "We have yet to see deterioration," Maguire says. "So far the improvement is sustained."

The injections themselves did cause complications in a few patients, such as a serious infection that resulted in permanent damage, and a dangerous increase in pressure in the eye. But there were no adverse reactions or any signs of problems associated with the gene therapy itself, the researchers reported.

While this disease is rare, the same approach could work for similar forms of genetic eye disease, Maguire says."There are a lot of retinal diseases like this, and if you added them together it's a big thing because they are all incurable."

If approved, the treatment would be marketed under the name Luxturna.

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First Gene Therapy For An Inherited Disorder Gets Expert ...

Scientists were understandably wary. Disabled AIDS viruses had not been used in human gene therapy. But I dont take no for an answer, Dr. Salzman said. I probably come just shy of stalking people.

The result of her lobbying was a tiny study in France in which researchers used a disabled form of HIV to deliver a normal form of the ALD gene. The investigators reported that the treatment seemed to stop brain degeneration in two boys.

Yet the idea behind the treatment seems almost preposterous: Take bone marrow stem cells from a boy with the ALD gene mutation. Insert a good gene into those cells and then infuse them back into the bone marrow.

Wait about a year while stem cells with the good genes multiply in the bone marrow. Eventually, they drift up into the brain, where they slowly turn into glial cells support cells that surround neurons and help insulate them.

The proper gene in the glial cells takes over, stopping the brain deterioration that would otherwise occur.

That unlikely process also explains why bone marrow transplants work, said David A. Williams, chief scientific officer at Boston Childrens Hospital and a principal investigator for the study. New bone marrow cells, from a healthy donor, supply good ALD genes to cells in the recipient that eventually become glial cells.

Either therapy must be administered early, before symptoms are apparent. In the year it takes for the treatment to become effective, the brains of children who are already showing symptoms can deteriorate to the point of no return.

The success of the small pilot study was enough to inspire the founding of a company, Bluebird Bio, which sponsored the bigger study in hopes of marketing gene therapy for ALD.

The company has now expanded that study to include an additional eight boys, and in separate research is following boys who had bone marrow transplants to compare outcomes.

For Paul Rojas of Dover Plains, N.Y., whose son was in the study, gene therapy has been a lifesaver. He never heard of the disease until his son Brandon, who was 7, started drooling, losing his ability to concentrate and listing to one side when he walked.

The diagnosis was a shock. And since Brandon was showing symptoms, it was too late for a bone-marrow transplant.

Brandons doctors, Mr. Rojas said, sat across from him and his wife, Liliana, in a small conference room and gave them the bad news: This is a disease that has no cure.

He had his 4-year-old, Brian, tested. He had the mutated gene, too.

The Rojases could not find a compatible donor for a bone-marrow transplant. But then they learned about the gene therapy trial and got Brian enrolled. He is now 7, with no sign of the disease.

But his older brother Brandon, now 10, no longer speaks, walks or eats. He has a feeding tube.

Brian misses playing with his brother, Mr. Rojas said. Brandon was his idol.

For Dr. Salzman, the results of the new gene therapy study have come too late. She had to get treatment for her son before he developed symptoms.

He had a cord blood transplant, which was successful. Her nephew also had one, but suffered complications and must use a wheelchair.

The results of the new study also give rise to a concern that is becoming a regular feature of gene therapy work and other new biotech therapies: How much will this treatment cost?

Bluebird Bio is not saying companies generally do not announce prices until their drugs are approved.

Dr. David A. Williams, chief scientific officer at Boston Childrens Hospital and a principal investigator of the new study, expects the price to be similar to the hundreds of thousands of dollars it costs for a bone-marrow transplant.

But the new treatment is a curative therapy, he said.

Dr. Friedmann is not assuaged by such arguments. The research enabling these products to come to market often begins with studies already paid for by grants from the federal government or from private foundations.

The expected prices, he said, are absolutely crazy.

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In a First, Gene Therapy Halts a Fatal Brain Disease - The ...

Virtually all cells in the human body contain genes, making them potential targets for gene therapy. However, these cells can be divided into two major categories: somatic cells (most cells of the body) or cells of the germline (eggs or sperm). In theory it is possible to transform either somatic cells or germ cells.

Gene therapy using germ line cells results in permanent changes that are passed down to subsequent generations. If done early in embryologic development, such as during preimplantation diagnosis and in vitro fertilization, the gene transfer could also occur in all cells of the developing embryo. The appeal of germ line gene therapy is its potential for offering a permanent therapeutic effect for all who inherit the target gene. Successful germ line therapies introduce the possibility of eliminating some diseases from a particular family, and ultimately from the population, forever. However, this also raises controversy. Some people view this type of therapy as unnatural, and liken it to "playing God." Others have concerns about the technical aspects. They worry that the genetic change propagated by germ line gene therapy may actually be deleterious and harmful, with the potential for unforeseen negative effects on future generations.

Somatic cells are nonreproductive. Somatic cell therapy is viewed as a more conservative, safer approach because it affects only the targeted cells in the patient, and is not passed on to future generations. In other words, the therapeutic effect ends with the individual who receives the therapy. However, this type of therapy presents unique problems of its own. Often the effects of somatic cell therapy are short-lived. Because the cells of most tissues ultimately die and are replaced by new cells, repeated treatments over the course of the individual's life span are required to maintain the therapeutic effect. Transporting the gene to the target cells or tissue is also problematic. Regardless of these difficulties, however, somatic cell gene therapy is appropriate and acceptable for many disorders, including cystic fibrosis, muscular dystrophy, cancer, and certain infectious diseases. Clinicians can even perform this therapy in utero, potentially correcting or treating a life-threatening disorder that may significantly impair a baby's health or development if not treated before birth.

In summary, the distinction is that the results of any somatic gene therapy are restricted to the actual patient and are not passed on to his or her children. All gene therapy to date on humans has been directed at somatic cells, whereas germline engineering in humans remains controversial and prohibited in for instance the European Union.

Somatic gene therapy can be broadly split into two categories:

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Overview of Gene Therapy Methods and Types of Gene Therapy

In BriefResearchers healed bone fractures by attracting stem cells to the area and injecting a mix of microbubbles and DNA encoding a bone protein at the break. This method could replace bone grafting for nonhealing fractures.

Fixing broken limb bones after serious injuries can challenge even the most skilled orthopedic surgeons. Too much bone loss makes regrowth impossible, and even smaller fractures make bone growth problematic if the patient is in poor health or at an advanced age.

When physicians encounter these kinds of nonhealing fractures, autologous bone grafts are the gold standard for treatment. These bone grafts involve harvesting a segment of healthy bone, typically from the pelvis of the patient, which is then used to bridge the portion of the break that isnt growing new bone adequately. However, bone grafts are not always possible, depending on the patients health and the extent of the damage from the break.

Some doctors in recent years have started to try something new: incorporating bone morphogenetic proteins (BMPs) into bone implants to enhance healing. This isnt a sure thing, though. Through their traditional administration, BMPs come with significant side effects including bone formation in soft tissues and bone resorption.

These side effects might haveoccurred because BMPs wereadministered in large doses, so researchers came up with a new strategy: use gene therapy to deliver not the protein itself, but the underlying gene instead. This way the cells will get BMP at physiological levels solely at the site of the injury.

However, gettinggene therapiesinto the right cells isnt always easy. The genes are typically delivered using viral vectors, and these come with their own safety concerns. The researchers in this case used a relatively new delivery mechanism instead: sonoporation.

In sonoporation, an ultrasound is used to cause gas-filled microbubbles with lipid shells to oscillate and create tiny, easily repaired holes in cells. These tiny holes allow DNA for gene therapy to enter into the right place without affecting other areas. The next step was ensuring that the gene therapy targeted the correctcells. The team targeteda special form of stem cells that can become bone cells and produce BMPs proficiently.

The researchers trialled their new strategy in broken pig shinbones and found that the technique healed fractures after a single dose. They first inserted collagen scaffolds, because they attract the stem cells, and then waited for two weeks to allow the scaffolds to recruit sufficient numbers of stem cells.

Next, they injected a mix of microbubbles and BMP-encoding DNA at the fracture site, and applied an ultrasound pulse. The team then waited for eight weeks after the single instance of the gene therapy. The experimental fractures were healed, while the control animals fractures were not.

This innovative therapy could improve the recovery of millions of people around the world. While human trials must be conducted before we know whether hospitals should adopt the procedure,many of its components have shown enough promise for scientists to utilize them insimilar bone-healing experiments: One fracture-fixing strategy incorporates a specific form of BPM, and another therapy uses stem cells to revitalize bone growth.

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Soon, Broken Bones Could be Fixed Using Gene Therapy and Microbubbles - Futurism

Boston Business Journal

Lexington biotech plots $86M IPO as key gene therapy trial nears Boston Business Journal A Lexington biotech developing gene therapy treatments for rare eye diseases has announced plans to raise up to $86 million in an initial public offering. Nightstar Therapeutics, a 23-employee company with a 3,300 square foot office in Lexington and a ... Nightstar files for $86M IPO to fund gene therapy trialsFierceBiotech all 14 news articles »

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Lexington biotech plots $86M IPO as key gene therapy trial nears - Boston Business Journal

If you listen closely to gut bacteria and host cells, you learn that they speak the same language. You might then pick up the language yourself, giving you the ability to join the microbiomehost conversation, which is known to have implications for human health. And if you ever had trouble being heard, you could try putting words in the mouths of all those jabbering bacteria, steering the microbiomehost conversation toward healthy conclusions.

When bacteria and host cells talk, they do so via signaling molecules, such as the ligands that interact with membrane-bound G-protein-coupled receptors (GPCRs). To keep an ear out for such ligands, scientists based at Rockefeller University and the Icahn School of Medicine at Mt. Sinai used the tools of bioinformatics and synthetic biology. These scientists, led by Sean Brady, Ph.D., director of Rockefeller University's Laboratory of Genetically Encoded Small Molecules, were particularly attuned to N-acyl amides, which interact with GPCR receptors.

Dr. Brady and colleagues, including co-investigator Louis Cohen, Ph.D., found that gut bacteria and human cells may not speak in the same dialect, but they can understand each other. Building on this observation, the scientists developed a method to genetically engineer the bacteria to produce molecules that have the potential to treat certain disorders by altering human metabolism. In a test of their system on mice, the introduction of modified gut bacteria led to reduced blood glucose levels and other metabolic changes in the animals.

Details of this work appeared August 30 in the journal Nature, in an article entitled Commensal Bacteria Make GPCR Ligands That Mimic Human Signalling Molecules. The article describes newly discovered commonalities in bacteria and host signaling, and it explains how these commonalities suggest ways gut flora could be engineered to have therapeutically beneficial effects on disease.

We found that N-acyl amide synthase genes are enriched in gastrointestinal bacteria and the lipids that they encode interact with GPCRs that regulate gastrointestinal tract physiology, wrote the articles authors. Mouse and cell-based models demonstrate that commensal GPR119 agonists regulate metabolic hormones and glucose homeostasis as efficiently as human ligands, although future studies are needed to define their potential physiological role in humans.

The language shared by bacteria and host cells involves the lock-and-key relationship of ligands, which bind to receptors on the membranes of human cells to produce specific biological effects. In this case, the bacteria-derived molecules are mimicking human ligands that bind to GPCRs. Many of the GPCRs are implicated in metabolic diseases, Dr. Brady noted, and are the most common targets of drug therapy. And they're conveniently present in the gastrointestinal tract, where the gut bacteria are also found.

"If you're going to talk to bacteria," explained Dr. Brady, "you're going to talk to them right there." (Gut bacteria are part of the microbiome, the larger community of microbes that exist in and on the human body.)

In its work, the team led by Drs. Cohen and Brady engineered gut bacteria to produce N-acyl amides that bind with a specific human receptor, GPR 119, which is known to be involved in the regulation of glucose and appetite and has previously been a therapeutic target for the treatment of diabetes and obesity. The bacterial ligands they created turned out to be almost identical structurally to the human ligands, said Dr. Cohen, an assistant professor of gastroenterology in the Icahn School of Medicine at Mt. Sinai.

Among the advantages of working with bacteria, continued Dr. Cohen, who spent five years in Dr. Brady's lab as part of Rockefeller's Clinical Scholars Program, is that their genes are easier to manipulate than human genes and much is already known about them. "All the genes for all the bacteria inside of us have been sequenced at some point," he pointed out.

Although the ligands are the product of nonhuman microorganisms, Dr. Brady says it's a mistake to think of the bacterial ligands they create in the lab as foreign. "The biggest change in thought in this field over the last 20 years is that our relationship with these bacteria isn't antagonistic," he commented. "They are a part of our physiology. What we're doing is tapping into the native system and manipulating it to our advantage."

"This is a first step in what we hope is a larger-scale, functional interrogation of what the molecules derived from microbes can do," Dr. Brady said. His plan is to systematically expand and define the chemistry that is being used by the bacteria in our guts to interact with us.

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Gene Therapy for the Bacteria of Our Microbiome Could Improve Our Health - Genetic Engineering & Biotechnology News