StemCell Therapy

Melissa Nolan

By the end of this chapter, students should be able to:

Those beautiful blue eyes you inherited from your mother are actually a result of a complex science known as Genetics. The scientific field of genetics studies genes in our DNA. Genes are units of heredity transferred from a parent to offspring and determine some characteristic of offspring. Your genes are responsible for coding all of your traits- including hair color, eye color, and so on. In recent years, scientists began exploring the concept of gene editing, which is the deliberate manipulation of genetic material to achieve desired results. Gene editing can potentially alter any given trait in an organism- from height to hair texture to susceptibility for certain diseases.

Gene editing applied to humans is referred to as Human Genetic Engineering, or HGE. There is extensive debate in and out of the scientific community regarding the ethics of HGE. Much of this debate stems from how this technology will affect society, and vice versa. Individuals may harbor concerns about the rise of designer babies or scientists playing God by determining the traits of an individual. On the contrary, HGE presents potential cures to diseases caused by genetic mutations. Human Genetic Engineering (HGE) is a novel technology which presents various ethical concerns and potential consequences. HGE should be approached cautiously and with extensive governmental regulation given its history, its current state, and the potential it has to change the world in the future.

Genetic Encoding of Proteins by MIT OpenCourseWare is licensed under CC BY-NC-SA 2.0

HGE utilizes CRISPR/Cas9 gene editing tools to cut out specific genes and replace them with a newly designed gene.

HGE encompasses a variety of methods which all work to produce a deliberate change in the human genome. The most common and prevalent way to edit the human genome is via CRISPR/Cas9. CRISPR stands for clustered regularly interspaced short palindromic repeats, and Cas9 is a protein that functions as scissors to cut DNA/genes. The CRISPR/Cas9 system originally developed as a part of a bacterias immune system, which can recognize repeats in DNA of invading viruses, then cut them out. Since then, scientists have harnessed the CRISPR/Cas9 system to cut DNA sequences of their choice and then insert new DNA sequences in their place.

The CRISPR/Cas9 system allows for designer genomes, and rapid engineering of any cells programming. With the use of CRISPR/Cas9, scientists can cut out certain traits from an individuals cells and insert new traits into those same cells.

CRISPR Cas9 System by Marius Walter is licensed under CC-BY-SA-4.0

Gene therapy is a recently-developed technology which can be applied to both somatic and germline genome editing.

Gene therapy concepts were initially introduced in the 1960s, utilizing outdated methods, such as recombinant DNA technology and viral vectors, to edit microorganisms genomes. Recombinant DNA consists of genetic material from multiple sources. The first experiments involved transferring a genome from one bacteria to another via a viral vector. Soon after was the first successful transformation of human cells with foreign DNA. The success of the experiment prompted public concern over the ethics of gene therapy, and led to political regulation. In the gene therapy report of the Presidents Commission in the United States, germline genome editing was deemed problematic over somatic genome editing. Also, non-medical genome editing was deemed problematic over medical genome editing. Germline genome editing occurs when scientists alter the genome of an embryo, so that the entire organism has altered genes and the traits can be passed to offspring. Somatic genome editing involves editing only a few cells in the entire organism so that traits can not be passed down to offspring. In response to the report, the rDNA Advisory Committee of the National Institutes of Health was formed and proposed the first guidelines for the gene therapy clinical trials. This is an example of technological determinism, in which technology determines the development of its social structure and cultural values or regulations.

In the past few decades, gene editing has advanced exponentially, introducing state-of-the-art technologies such as the CRISPR/Cas9 system, which was developed to induce gene modifications at very specific target sites. Thus, gene editing became a major focus for medical research (Tamura, 2020). Gene editing has led to the potential for development of treatment strategies for a variety of diseases and cancers. So far, somatic genome editing has shown promise in treating leukemia, melanoma, and a variety of other diseases. In this way, HGE may be demonstrative of cultural determinism, in which the culture we are raised presents certain issues which necessitate the development of a specific technology.

DNA CRISPR Scissors by Max Pixel is licensed under CC0 1.0

CRISPR/Cas9 is the primary technology proposed for use in HGE. HGE presents a variety of pros and cons to society.

Somatic genome editing in HGE via the CRISPR/Cas9 system has proven to be effective at editing specific genome sites. Since 2015, genome editing technologies have been used in over 30 human clinical trials and have shown positive patient outcomes. The treatment of disease may be a positive benefit of HGE, but there are also various potential risks. Various forms of deliberative democracies formed in recent years to address scientific and ethical concerns in HGE. Deliberative democracies afrm the need to justify technological decisions made by citizens and their representatives with experts in the field via deliberation. Overall, the consensus remains that the pros and cons of HGE are not equivalent enough to justify widespread use of the technology.

Current human clinical trials show successful transformation of human immune cells to HIV-resistant cells. This implies that HGE may be the cure for HIV(Hu, 2019). Other successful somatic genome editing trials treated myeloma, leukemia, sickle cell disease, various forms of epithelial cancers, and hemophilia. Thus, gene editing has provided novel treatment options for congenital diseases and cancers (Tamaura, 2020). Congenital diseases are those present from birth, and typically have a genetic cause. For these reasons, scientific summits concluded HGE is ethical for research regarding somatic genome editing in congenital diseases and cancers.

There are many safety concerns regarding CRISPR applications, mainly in germline genome editing. As a result of technological determinism, a leading group of CRISPR/Cas9 scientists and ethicists met for the international Summit on Human Gene Editing. The summit determined that heritable genome research trials may be permitted only following extensive research on risks and benefits of HGE. However, the summit concluded that federal funding cannot be used to support research involving human embryos with germline editing techniques. These decisions were made to avoid potential risks such as the following.

The major concerns regarding germline genome editing in HGE include: serious injury or disability, a blurry line between therapeutic applications of HGE and medical applications, misapplications, potential for eugenics ( the study of how to arrange reproduction within a human population to increase the occurrence of heritable characteristics regarded as desirable), and inequitable access to the technology.

HGE is a complex technology which presents a variety of risk factors for the coming decades. Deliberative democracy is necessary to keep this technology in check, ethically.

The future of HGE is uncertain and requires immense forethought. The American Society of Human Genetics workgroup developed a position statement on human germline engineering. The statement argues that it is inappropriate to perform germline gene editing that culminates in human pregnancy; and that in vitro(outside of an organism) germline editing should be permitted with appropriate oversight. It also states future clinical human germline editing requires ethical justification, compelling medical rationale, and evidence that supports its clinical usage. Many of these decisions were made based on the potential concerts over the future possibilities of the technology.

At the societal level, there may be concerns related to eugenics, social justice, and accessibility to technology. Eugenics could potentially reinforce prejudice and enforce exclusivity in certain physical traits. Traits can be preselected for, thus labeling some as good and others as unfavorable. This may perpetuate existing racist ideals, for example.

Moreover, germline genome editing may also increase the amount of inequality in a society. Human germline editing is likely to be very expensive and access may be limited to certain geographic regions, health systems, or socioeconomic statuses. Even if human genetic engineering is only used for medical purposes, genetic disease could become an artifact of class, location, or ethnic group. Therefore, preclinical trials are necessary to establish validity, safety, and efficacy before any wide scale studies are initiated.

Others argue that HGE may lessen genetic diversity in a human population, creating a biological monoculture that could lead to disease susceptibility and eventual extinction. Analyses have predicted that there will be negligible effect on diversity and will more likely ensure the health and longevity of humans (Russel, 2010). Legacy thinking may be responsible for the hesitations towards continuing forward with HGE, as there are also many potential pros for genetic engineering. Legacy thinking is using outdated thinking strategies and actions which may not be useful anymore.

In an alternative modernity, we can imagine HGE as an end-all for most congenital diseases and cancers. Moreover, it may be used in germline gene editing to prevent certain birth defects or heritable diseases. So, although HGE has a variety of potential risk factors, there is also great promise for novel medical therapies in the coming decades. The continued use of this technology should be approached cautiously and with extensive governmental regulation, allowing for research regarding its medical applications only.

In 2016, germline gene editing was proven feasible and effective in chickens by leading researchers in genetic engineering, Dimitrov and colleagues. In this study, scientists used CRISPR/Cas9 to target the gene for an antibody/ immunoglobulin commonly produced in chickens. Antibodies are proteins produced in immune response. In the resulting population, the chickens grew normally and healthily with modified antibodies which conferred drug resistance. This study was the first to prove that germline editing is both feasible and effective.

HGE is a rapidly expanding field of research which presents novel possibilities for the coming decades. HGE utilizes CRISPR/Cas9 gene editing tools to cut out specific genes and replace them with a newly designed gene. As important as this technology is, it is also important to recognize how new it is. Gene therapy research began in the 1960s, with somatic cell editing only commencing in the past two decades. This has presented many advantages for the potential treatment of congenital diseases, but also presents various risks. Those risks stem from germline gene editing and include eugenics and inequitable access to the technology creating large socio economic divides. In the future, more regulation should be placed on the advancement of HGE research before larger-scale studies take place.

1. What is the primary technology proposed for use in HGE?

A. Recombinant DNA technology

B. CRISPR/Cas9

C. Bacterial Transformation

D. Immunoglobulin

2. When was gene therapy concepts first introduced?

A. 1920s

B. 1940s

C. 1960s

D. 1980s

3. What is a major ethical concern regarding HGE addressed in this chapter?

A. Potential for ageism

B. Gene editing is only 50% effective

C. HGE can only be used in Caucasians

D. Potential for eugenics

Answers:

Baltimore, D. et. al.(2015). A prudent path forward for genomic engineering and germline gene modification. Science. https://doi.org/10.1126/science.aab1028

Brokowski, C., & Adli, M. (2019). CRISPR Ethics: Moral Considerations for Applications of a Powerful Tool. Journal of Molecular Biology. https://doi.org/10.1016/j.jmb.2018.05.044

Cong, L., Ran, F., & Zhang, F. (2013). Multiplex Genome Engineering Using CRISPR/Cas9 Systems. Science. https://doi.org/10.1126/science.1231143

Dimitrov, L., et. al. (2016). Germline Gene Editing in Chickens by Efficient CRISPR-Mediated Homologous Recombination in Primordial Germ Cells. Plos One. https://doi.org/10.1371/journal.pone.0154303

Hu, C. (2019). Safety of Transplantation of CRISPR CCR5 Modified CD34+ Cells in HIV-Infected Subjects with Hematological Malignancies. U.S National Library of Medicine. https://clinicaltrials.gov/ct2/show/NCT03164135

Ormond, K., et. al.(2017). Human Germline Genome Editing. AJHG. https://doi.org/10.1016/j.ajhg.2017.06.012

Russell P.(2010) The Evolutionary Biological Implications of Human Genetic Engineering, The Journal of Medicine and Philosophy: A Forum for Bioethics and Philosophy of Medicine. https://doi.org/10.1093/jmp/jhq004

Tamura, R., & Toda, M. (2020). Historic Overview of Genetic Engineering Technologies for Human Gene Therapy. Neurologia medico-chirurgica. https://doi.org/10.2176/nmc.ra.2020-0049

Thomas, C. (2020). CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy for Relapsed/Refractory B Cell Non-Hodgkin Lymphoma. ClinicalTrials. https://clinicaltrials.gov/show/NCT04637763

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18 Human Genetic Engineering - Clemson University

Genetic engineering is the process of altering the genetic composition of plants, animals, and humans. The most practical application of genetic engineering is to create a more sustainable food system for the people of Earth, but there are other ways we can use it to our advantage as well.

Unfortunately, there are both pros and cons of genetic engineering. For every benefit, there is a list of concerns and potential problems we need to consider. There is a substantive argument on both sides of genetic engineering, and well explore both ahead.

Most people tend to focus on the negatives of genetic engineering, but there are some substantial positive we need to consider as well. Genetic engineering is a debate, and there are some good points on each side. You have to look at both the pros and cons of genetic engineering if you want to make an informed decision on the matter.

Evolution takes thousands of years to adapt to our surroundings, but genetic engineering offers a quicker path forward. With the assistance of genetic engineering, we could force our bodies to adapt to the changing climate of our planet.

Additionally, we could tack-on some extra years to our lives by altering our cells, so our bodies dont deteriorate as quickly as they currently do. The fountain of youth might be within our reach, and many look forward to advancements in the area of genetic engineering.

If we choose to go down this path, well feel better as we age and be able to outlast some of the diseases that currently take us down. We still wont be able to live forever, but genetic engineering shows promise in extending the prime of our lives.

Food shortage is a massive problem in the world, especially with the growing population. Were destroying natural habitats to make way for farmland, and overgrazing is causing current pastures to become dry and uninhabited.

The answer to this problem could come in the form of genetic engineering. If we can alter the composition of vegetables and animals, we can create new foods that might have more nutritional value than nature creates on its own.

We might even be able to advance to a point where foods give us medicines we need to combat widespread viruses and illnesses. Food is one of the most promising spaces when considering the prospect of genetic engineering.

A lot of diseases depend on genetic predisposition. Some people are more likely to get cancer, Alzheimers and other diseases than their neighbor. With genetic engineering, we can get rid of these genetic predispositions once and for all.

There will likely still be some environmental concerns that will cause diseases, but if we start altering the genes of humans, we may become resistant to genetic abnormalities. Family history wont mean anything when it comes to things like cancer, and we can start eliminating diseases that are completely based on genetics.

There are already a handful of diseases and illnesses we can detect while a baby is still in the womb. We even can genetically engineer some diseases and illnesses out of a babys system before theyre born.

Finding out your baby has a disease can be devastating, and some parents make the difficult choice to spare their child possible pain. If you know that your baby might suffer and die a few months after theyre born, you have to decide whether or not you want to roll the dice.

In the future, we might be able to eliminate the chances of unhealthy babies. Diseases like Huntingtons offer a substantial chance that the carrier will pass it onto their child. If the child isnt positive for the disease, theyll still be a carrier and have to deal with the same dilemma when it comes time to have kids of their own.

Genetic engineering has the potential to stop these threats in their tracks. Parents wont have to worry about birthing a healthy son or daughter. Science will guarantee that every baby is happy and healthy when they come into this world.

Of course, genetic engineering isnt entirely positive. There is an upside to the ability to genetically alter humans and animals, but only in ideal situations.

Our world isnt perfect, and scientists make mistakes all the time. We cant assume that genetic engineering will be available to the entirety of the human population, which is a flaw in itself.

The negatives of genetic engineering seem to outweigh the positives, especially since there is so much room for error. We dont know what were tampering with, which opens the door to a host of potential problems.

There are a couple of ethical problems with genetic engineering that we need to consider as a society. Those who subscribe to religion will see genetic engineering as blasphemy, for instance. Wed be playing God, in a sense. Anyone who believes in creation will be expressly against genetic engineering especially in human children.

Those who are on the opposite side of the spectrum from religious people probably wont love genetic engineering either. Genetically engineered food might work, but changing the genes of people will add to the overpopulation problem were currently experiencing.

Diseases are one of the most effective forms of population control. We dont have the heart to eliminate other humans in the name of population control, so disease does it for us. If we eliminate diseases, humans will have virtually no threat left on this planet.

Living longer lives might be ideal, but it isnt practical. If we extend the prime of our lives, were opening the door to having more children. Since all children would be in perfect health, well see a population increase that could have devastating consequences.

If genetic engineering becomes a reality, it will likely only be available to the richest members of society. Theyll be able to extend their lives, limit diseases, and make sure their children are always healthy when theyre born

When this happens, natural selection is completely obsolete. Instead, the wealthiest in society will thrive while the poor will die-out. Eventually, genetic diversity will completely disappear as genetically engineered children all express the most desirable characteristics

This problem also arises in nature if we decide to engineer plants and animals genetically. These organisms might start as food, but could introduce themselves to the wild and take over. Theyll decimate natural species, and eventually be the only thing left.

One of the biggest hurdles in genetic engineering is the possibility of errors or genetic defects, especially in humans. Scientists have a general understanding of what creates a functioning human, but they dont yet have all the pieces to the puzzle.

When it comes down to changing humans at a cellular level, scientists dont yet have the understanding of how small changes can affect the development of a growing baby. Changing genes could result in more damaging birth defects or even miscarriages.

Furthermore, tampering with diseases could end up creating a super-disease that is even harder to combat. There are too many variables in the human body for genetic engineering to work to the fullest potential. Even if it could, people will probably be too nervous to trust scientists tampering with the cells of their future children.

Science still isnt at a point where they can alter the genes of humans to prevent all diseases in unborn children, but it might be there soon. When that time comes, some might take genetic engineering to its logical extreme.

Our priority will be to create healthy children. Once we perfect this process, though, where to, we go? The next logical step is the ability to pick certain traits that our children will have. We might be able to select whether we have a boy or girl. Then, we can decide what eye color and hair color they have.

Pretty soon, were selecting every trait that our child has before they leave the womb. Nature will be virtually out of the question at this point, and people with enough money will design their babies from scratch.

Since the pros and cons of genetic engineering are compelling, its worth it to explore the possibility further. We still havent reached a place where scientists fully understand the opportunities genetic engineering presents, so they still have years of research on their hands.

In the end, though, no system of genetically altering humans, animals, or plants will be perfect. There is a massive potential for errors, and we likely wont have equal opportunities if and when scientists ever crack the case.

Although the positives of genetic engineering are convincing, the negatives can be terrifying. If we ever get to the point where we can genetically alter humans, we need to consider the moral, ethical, and practical application of technology before going any further.

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Pros and Cons of Genetic Engineering - Benefits and Risks

How artificial skin is made and its uses, from treating burns to skin cancer  South China Morning Post

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How artificial skin is made and its uses, from treating burns to skin cancer - South China Morning Post

BIORESTORATIVE THERAPIES, INC. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS. (form 10-K)  Marketscreener.com

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BIORESTORATIVE THERAPIES, INC. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS. (form 10-K) - Marketscreener.com

Why do scientists study the genes of other organisms?

All living things evolved from a common ancestor. Therefore, humans, animals, and other organisms share many of the same genes, and the molecules made from them function in similar ways.

Scientists have found many genes that have been preserved through millions of years of evolution and are present in a range of organisms living today. They can study these preserved genes and compare the genomes of different species to uncover similarities and differences that improve their understanding of how human genes function and are controlled. This knowledge helps researchers develop new strategies to treat and prevent human disease. Scientists also study the genes of bacteria, viruses, and fungi for solutions to prevent or treat infection. Increasingly, these studies are offering insight into how microbes on and in the body affect our health, sometimes in beneficial ways.

Increasingly sophisticated tools and techniques are allowing NIGMS-funded scientists to ask more precise questions about the genetic basis of biology. For example, theyre studying the factors that control when genes are active, the mechanisms DNA uses to repair broken or damaged segments, and the complex ways traits are passed to future generations. Another focus of exploration involves tracing genetic variation over time to detail human evolutionary history and to pinpoint the emergence of disease-related attributes. These areas of basic research will continue to build a strong foundation for more disease-targeted studies.

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Genetics - National Institute of General Medical Sciences (NIGMS)

Almost every human trait and disease has a genetic component, whether inherited orinfluenced by behavioral factors such as exercise. Genetic components can also modifythe bodys response to environmental factors such as toxins. Understanding theunderlying concepts of human genetics and the role of genes, behavior, and theenvironment is important for appropriately collecting and applying genetic and genomicinformation and technologies during clinical care. It is important in improving diseasediagnosis and treatment as well. This chapter provides fundamental information aboutbasic genetics concepts, including cell structure, the molecular and biochemical basisof disease, major types of genetic disease, laws of inheritance, and the impact ofgenetic variation.

Cells are the fundamental structural and functional units of every known livingorganism. Instructions needed to direct activities are contained within a DNA(deoxyribonucleic acid) sequence. DNA from all organisms is made up of the samechemical units (bases) called adenine, thymine, guanine, and cytosine, abbreviatedas A, T, G, and C. In complementary DNA strands, A matches with T, and C with G, toform base pairs. The human genome (total composition of genetic material within acell) is packaged into larger units known as chromosomesphysically separatemolecules that range in length from about 50 to 250 million base pairs. Human cellscontain two sets of chromosomes, one set inherited from each parent. Each cellnormally contains 23 pairs of chromosomes, which consist of 22 autosomes (numbered 1through 22) and one pair of sex chromosomes (XX or XY). However, sperm and ovanormally contain half as much genetic material: only one copy of eachchromosome.

Each chromosome contains many genes, the basic physical and functional units ofheredity. Genes are specific sequences of bases that encode instructions for how tomake proteins. The DNA sequence is the particular side-by-side arrangement of basesalong the DNA strand (e.g., ATTCCGGA). Each gene has a unique DNA sequence. Genescomprise only about 29 percent of the human genome; the remainder consists ofnon-coding regions, whose functions may include providing chromosomal structuralintegrity and regulating where, when, and in what quantity proteins are made. Thehuman genome is estimated to contain 20,000 to 25,000 genes.

Although each cell contains a full complement of DNA, cells use genes selectively.For example, the genes active in a liver cell differ from the genes active in abrain cell because each cell performs different functions and, therefore, requiresdifferent proteins. Different genes can also be activated during development or inresponse to environmental stimuli such as an infection or stress.

Many, if not most, diseases are caused or influenced by genetics. Genes, through theproteins they encode, determine how efficiently foods and chemicals are metabolized,how effectively toxins are detoxified, and how vigorously infections are targeted.Genetic diseases can be categorized into three major groups: single-gene,chromosomal, and multifactorial.

Changes in the DNA sequence of single genes, also known as mutations, cause thousandsof diseases. A gene can mutate in many ways, resulting in an altered protein productthat is unable to perform its normal function. The most common gene mutationinvolves a change or misspelling in a single base in the DNA.Other mutations include the loss (deletion) or gain (duplication or insertion) of asingle or multiple base(s). The altered protein product may still retain some normalfunction, but at a reduced capacity. In other cases, the protein may be totallydisabled by the mutation or gain an entirely new, but damaging, function. Theoutcome of a particular mutation depends not only on how it alters aproteins function, but also on how vital that particular protein is tosurvival. Other mutations, called polymorphisms, are natural variations in DNAsequence that have no adverse effects and are simply differences amongindividuals.

In addition to mutations in single genes, genetic diseases can be caused by largermutations in chromosomes. Chromosomal abnormalities may result from either the totalnumber of chromosomes differing from the usual amount or the physical structure of achromosome differing from the usual structure. The most common type of chromosomalabnormality is known as aneuploidy, an abnormal number of chromosomes due to anextra or missing chromosome. A usual karyotype (complete chromosome set) contains 46chromosomes including an XX (female) or an XY (male) sex chromosome pair. Structuralchromosomal abnormalities include deletions, duplications, insertions, inversions,or translocations of a chromosome segment. (See Appendix F for more information aboutchromosomal abnormalities.)

Multifactorial diseases are caused by a complex combination of genetic, behavioral,and environmental factors. Examples of these conditions include spina bifida,diabetes, and heart disease. Although multifactorial diseases can recur in families,some mutations such as cancer can be acquired throughout an individualslifetime. All genes work in the context of environment and behavior. Alterations inbehavior or the environment such as diet, exercise, exposure to toxic agents, ormedications can all influence genetic traits.

The basic laws of inheritance are useful in understanding patterns of diseasetransmission. Single-gene diseases are usually inherited in one of several patterns,depending on the location of the gene (e.g., chromosomes 1-22 or X and Y) andwhether one or two normal copies of the gene are needed for normal protein activity.Five basic modes of inheritance for single-gene diseases exist: autosomal dominant,autosomal recessive, X-linked dominant, X-linked recessive, and mitochondria. (Seediagram on following page.)

All individuals are 99.9 percent the same genetically. The differences in thesequence of DNA among individuals, or genetic variation, explain some of thedifferences among people such as physical traits and higher or lower risk forcertain diseases. Mutations and polymorphisms are forms of genetic variation. Whilemutations are generally associated with disease and are relatively rare,polymorphisms are more frequent and their clinical significance is not asstraightforward. Single nucleotide polymorphisms (SNPs, pronouncedsnips) are DNA sequence variations that occur when a singlenucleotide is altered. SNPs occur every 100 to 300 bases along the 3 billion-basehuman genome. A single individual may carry millions of SNPs.

Although some genetic variations may cause or modify disease risk, other changes mayresult in no increased risk or a neutral presentation. For example, genetic variantsin a single gene account for the different blood types: A, B, AB, and O.Understanding the clinical significance of genetic variation is a complicatedprocess because of our limited knowledge of which genes are involved in a disease orcondition and the multiple gene-gene and gene-behavior-environment interactionslikely to be involved in complex, chronic diseases. New technologies are enablingfaster and more accurate detection of genetic variants in hundreds or thousands ofgenes in a single process.

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GENETICS 101 - Understanding Genetics - NCBI Bookshelf

People always think Im skinny because of good genetics theyre shocked when they see what I used to lo...  The US Sun

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Procedure to Surgery for Arthritis Is Recommended After First Failed Non-Operative Therapy  DocWire News

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Procedure to Surgery for Arthritis Is Recommended After First Failed Non-Operative Therapy - DocWire News

OverviewYour immune system is made of up a complex collection of cells and organs. The system works together to protect you from germs and helps you get better when you get sick.What is the immune system?

Your immune system is a large network of organs, white blood cells, proteins (antibodies) and chemicals. This system works together to protect you from foreign invaders (bacteria, viruses, parasites, and fungi) that cause infection, illness and disease.

Your immune system works hard to keep you healthy. Its job is to keep germs out of your body, destroy them or limit the extent of their harm if they get in.

When your immune system is working properly: When your immune system is working properly, it can tell which cells are yours and which substances are foreign to your body. It activates, mobilizes, attacks and kills foreign invader germs that can cause you harm. Your immune system learns about germs after youve been exposed to them too. Your body develops antibodies to protect you from those specific germs. An example of this concept occurs when you get a vaccine. Your immune system builds up antibodies to foreign cells in the vaccine and will quickly remember these foreign cells and destroy them if you are exposed to them in the future. Sometimes doctors can prescribe antibiotics to help your immune system if you get sick. But antibiotics only kill certain bacteria. They dont kill viruses.

When your immune system is not working properly: When your immune system cant mount a winning attack against an invader, a problem, such as an infection, develops. Also, sometimes your immune system mounts an attack when there is no invader or doesnt stop an attack after the invader has been killed. These activities result in such problems as autoimmune diseases and allergic reactions.

Your immune system is made of up a complex collection of cells and organs. They all work together to protect you from germs and help you get better when youre sick. The main parts of the immune system are:

Many deficiencies and disorders can damage or disrupt your immune system. Some medicines make it harder for your body to fight infection. Certain health conditions cause your immune system to attack healthy cells or make it hard for your immune system to protect you from harmful germs. They include:

Just like the rest of your body, your immune system needs nourishment, rest, and a healthy environment to stay strong. Certain lifestyle changes can boost your immune system and help you avoid illness. To keep your immune system running smoothly, you should:

If you feel like youre always sick or you have symptoms that never seem to go away, you should visit your doctor. Some symptoms could be signs of an autoimmune disease. These symptoms include:

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Immune System: Parts & Common Problems - Cleveland Clinic

Your immune system is your bodys defense againstinfections and other harmfulinvaders. Without it, you would constantly get sick frombacteria or viruses.

Your immune system is made up of special cells, tissues, and organs that work together to protect you.

The lymph, or lymphatic, system is a major part of the immune system. It's a network of lymph nodes and vessels. Lymphatic vessels are thin tubes that branch, like blood vessels,throughout the body. They carry a clear fluid called lymph. Lymph contains tissue fluid, waste products, and immune system cells. Lymph nodes are small, bean-shaped clumps of immune system cells that are connected by lymphatic vessels. They contain white blood cells that trap viruses, bacteria, and other invaders, including cancer cells.

White blood cells are the cells of the immune system. They are made in one of your lymph organs, the bone marrow. Other lymph organs include thespleen and thymus.

When your immune system doesn't work the way it should, it is called an immune system disorder. You may:

Beborn with a weak immune system. This is called primary immune deficiency.

Get a disease that weakens your immune system. This is called acquired immune deficiency.

Have animmune system that is too active.This may happen with an allergic reaction.

Have animmune system thatturns against you. This is called autoimmune disease.

Here are some common examples:

Severe combined immunodeficiency (SCID).This is an example of an immune deficiency that is present at birth.Children arein constant danger of infections from bacteria, viruses, and fungi. This disorder is sometimes called bubble boy disease.In the 1970s, a boyhad to live in a sterile environment inside a plastic bubble. Children with SCID are missing important white blood cells.

Temporary acquired immune deficiencies.Yourimmune system can be weakened by certain medicines, for example. This canhappen to peopleon chemotherapy or other drugs used to treat cancer. It can also happen to people followingorgan transplants who take medicine to prevent organ rejection.Also, infections like the flu virus, mono (mononucleosis), and measlescan weaken the immune system for a brief time. Your immune system can also be weakened by smoking, alcohol,and poor nutrition.

AIDS.HIV, which causes AIDS, is an acquired viralinfection that destroys important white blood cells and weakens the immune system.People withHIV/AIDS become seriously ill with infections that most peoplecan fight off. These infections are called opportunistic infections because they take advantage of weak immune systems.

If you are born with certain genes, your immune system may react to substances inthe environment that are normally harmless. These substances are called allergens. Having an allergic reaction is the most common example of an overactive immune system. Dust, mold, pollen,and foods are examples of allergens.

Some conditions caused by an overactive immune system are:

Asthma.The response in your lungs can cause coughing, wheezing, and trouble breathing. Asthma can be triggered by common allergens like dust or pollenor by an irritant like tobacco smoke.

Eczema.An allergen causes an itchy rash known as atopic dermatitis.

Allergic rhinitis.Sneezing, a runny nose, sniffling, and swelling of your nasal passages from indoor allergens like dust and pets or outdoor allergens like pollens or molds.

Inautoimmune diseases, the body attacks normal, healthy tissues. The cause is unknown. It is probably a combination of a persons genes and something in the environmentthat triggers those genes.

Three common autoimmune diseases are:

Type 1 diabetes.The immune systemattacksthe cells inthe pancreas that make insulin. Insulin removes sugar fromthe blood to use as energy.

Rheumatoid arthritis.Thistype of arthritiscauses swelling and deformities of the joints. An auto-antibody called rheumatoid factoris in the blood of some people with rheumatoid arthritis.

Lupus.This disease that attacks body tissues, including thelungs, kidneys, and skin. Many types of auto-antibodies are found in the blood ofpeople with lupus.

No one knows exactly what causes autoimmune diseases, but many factors seem to be involved. If you have an immune system disorder, learn as much as you can aboutit. And work closely with yourhealthcare providersto manage it.

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Disorders of the Immune System | Johns Hopkins Medicine

What is biotechnology?

Biotechnology is the use of biology to develop new products, methods and organisms intended to improve human health and society. Biotechnology, often referred to as biotech, has existed since the beginning of civilization with the domestication of plants, animals and the discovery of fermentation.

Early applications of biotech led to the development of products such as bread and vaccines. However, the discipline has evolved significantly over the last century in ways that manipulate the genetic structures and biomolecular processes of living organisms. The modern practice of biotechnology draws from various disciplines of science and technology, including the following:

This approach has resulted in innovations and breakthroughs in the following areas:

Modern applications of biotechnology work most often through genetic engineering, which is also known as recombinant DNA technology. Genetic engineering works by modifying or interacting with the genetic cell structures. Every cell in an animal or plant contains genes that produce proteins. It's those proteins that determine the characteristics of the organism.

By modifying or interacting with genes, scientists can strengthen the characteristics of an organism or create an entirely new organism. These modified and new organisms may be beneficial to humans, such as crops with higher yields or increased resistance to drought. Genetic engineering also enables the genetic modification and cloning of animals, two controversial developments.

Biotechnology began at least 6,000 years ago with the agricultural revolution. This early era was characterized by exploiting living organisms in their natural forms or modifying their genetic makeup through selective breeding.

Around the same time, humans learned to harness the biological process of fermentation to produce bread, alcohol and cheese. People also began changing the genetic makeup of domesticated plants and animals through selective breeding.

Selective breeding works by breeding parents with desirable characteristics to express or eliminate certain genetic characteristics in their offspring. Over time, species that are selectively bred evolve to be different from their wild ancestors. For instance, during the agricultural revolution, wheat was selectively bred to stay on its stem when harvested instead of falling to the ground like wild wheat. Dogs were selectively bred to be more docile than their wolf ancestors.

However, biotech methods such as selective breeding can take a long time to show changes in species. Biotechnology remained limited to these slow, agricultural methods until the 19th century when biologist Gregor Mendel discovered the basic principles of heredity and genetics.

Also, during that era, scientists Louis Pasteur and Joseph Lister discovered the microbial processes of fermentation. This laid the foundation for biotechnology industries where scientists interact more directly with the molecular and genetic processes of organisms.

Based on the work of these scientists, genetic engineering was developed in 1973. This method is the foundation of modern biotechnology practices and recent advances. It enabled the first direct manipulation of plant and animal genomes, which is the complete set of genes present in a cell.

Over the last 100 hundred years or so, biotechnology emerged with the following discoveries and advancements:

1919. Hungarian scientist Karl Ereky coins the term biotechnology.

1928. Alexander Fleming discovers penicillin, the first true antibiotic.

1943. Oswald Avery proves DNA carries genetic information.

1953. James Watson and Francis Crick discover the double helix structure of DNA.

1960s. Insulin is synthesized to fight diabetes, and vaccines for measles, mumps and rubella are developed.

1969. The first synthesis of an enzyme in vitro, or outside the body, is conducted.

1973. Herbert Boyer and Stanley Cohen develop genetic engineering with the first insertion of DNA from one bacteria into another.

1980s. The first biotech drugs to treat cancer are developed.

1890. The United States Supreme Court rules that a "live human-made microorganism is patentable subject matter," meaning GMOs can be intellectual property.

1982. A biotech-developed form of insulin becomes the first genetically engineered product approved by the U.S. Food and Drug Administration (FDA).

1983. The first genetically modified plant is introduced.

1993. GMOs are introduced into agriculture with the FDA approval of growth hormones that produce more milk in cows.

1997. The first mammal is cloned.

1998. The first draft of the Human Genome Project is created, giving scientists access to over 30,000 human genes and facilitating research on treatment of diseases such as cancer and Alzheimer's.

2010. The first synthetic cell is created.

2013. The first bionic eye is created.

2020. MRNA vaccine and monoclonal antibody technology is used to treat the SARS-CoV-2 virus.

The science of biotechnology is broken down into subdisciplines that are color-coded based on common uses and applications.

The use and commercialization of modern biotechnology often fall into four main fields: environment, medicine, industry and agriculture.

The aim of environmental biotechnology is to develop sustainable environmental practices that reduce pollution and waste. The following are examples of environmental biotech:

Medical biotechnology, also known as biopharma, aims to fight and prevent disease and improve healthcare. Biotechnology and biomedical research are the basis of the modern pharmaceutical industry. Uses include the following:

Industrial biotechnology involves using microorganisms to produce industrial goods. Examples include the following:

Agricultural biotechnology genetically engineers plants and animals to produce more efficient agriculture, increase nutritional value and reduce food insecurity. Some examples of agricultural biotechnology are the following:

Biotechnology production offers a variety of advantages and solutions to critical problems. The main ones are the following:

Biotechnology also comes with disadvantages and misuse. The main disadvantages include the following:

Concerns about biotechnology's disadvantages have led to efforts to enact legislation restricting or banning certain processes or programs, such as human cloning, GMOs and embryonic stem-cell research.

Biotechnology is critical to environmentally sound advancements in agriculture. Learn more about how technology like artificial intelligence (AI) is improving the food industry.

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What is Biotechnology? Definition, Types and Applications | TechTarget

Where Does Novavax Inc (NVAX) Stock Fall in the Biotechnology Field After It Is Lower By -12.99% This Week?  InvestorsObserver

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Where Does Novavax Inc (NVAX) Stock Fall in the Biotechnology Field After It Is Lower By -12.99% This Week? - InvestorsObserver

Should Biotechnology Stock Outlook Therapeutics Inc (OTLK) Be in Your Portfolio Thursday?  InvestorsObserver

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Should Biotechnology Stock Outlook Therapeutics Inc (OTLK) Be in Your Portfolio Thursday? - InvestorsObserver

SANA BIOTECHNOLOGY, INC. : Results of Operations and Financial Condition, Financial Statements and Exhibits (form 8-K)  Marketscreener.com

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SANA BIOTECHNOLOGY, INC. : Results of Operations and Financial Condition, Financial Statements and Exhibits (form 8-K) - Marketscreener.com

Genetic therapies aim to treat or cure conditions by correcting problems in your DNA. Your DNA, including specific genes, contains instructions for making proteins that are essential for good health. Mutations, or changes in your DNA, can lead to proteins that do not work properly or that are missing altogether. These changes can cause genetic, or inherited, disorders such as cystic fibrosis, thalassemia, hemophilia, and sickle cell disease.

Genetic therapies are approaches that treat genetic disorders by providing new DNA to certain cells or correcting the DNA. Gene transfer approaches, also called gene addition, restore the missing function of a faulty or missing gene by adding a new gene to affected cells. The new gene may be a normal version of the faulty gene or a different gene that bypasses the problem and improves the way the cell works.

Genome editing is a newer approach that allows precise correction or other targeted changes to the DNA in cells to restore a cells function. Genome editing can:

Gene transfer or genome editing treatments can directly modify the cells in your body, or your cells can be collected and treated outside of your body and then returned to you. For example, a doctor can remove immune system cells, cells that are part of your bodys natural defense system, or bone marrow cells from your body, modify their DNA, and then re-introduce them to your body.

The only genetic therapies that are currently approved by the U.S. Food and Drug Administration (FDA) are for a rare inherited eye condition, as well as certain types of cancer. Genetic therapies that are in development could treat or cure other inherited disorders; treat other cancers; or treat infections, including HIV.

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Genetic Therapies - What Are Genetic Therapies? | NHLBI, NIH

Espaol

The genes in your bodys cells play a key role in your health. Indeed, a defective gene or genes can make you sick.

Recognizing this, scientists have worked for decades on ways to modify genes or replace faulty genes with healthy ones to treat, cure, or prevent a disease or medical condition.

This research is paying off, as advancements in science and technology today are changing the way we define disease, develop drugs, and prescribe treatments.

The U.S. Food and Drug Administration has approved multiple gene therapy products for cancer and rare disease indications.

Genes and cells are intimately related. Within the cells of our bodies, there are thousands of genes that provide the information to produce specific proteins that help make up the cells. Cells are the basic building blocks of all living things; the human body is composed of trillions of them.

The genes provide the information that makes different cells do different things. Groups of many cells make up the tissues and organs of the body, including muscles, bones, and blood. The tissues and organs in turn support all our bodys functions.

Sometimes the whole or part of a gene is defective or missing from birth. This is typically referred to as a genetically inherited mutation.

In addition, healthy genes can change (mutate) over the course of our lives. These acquired mutations can be caused by environmental exposures. The good news is that most of these genetic changes (mutations) do not cause disease. But some inherited and acquired mutations can cause developmental disorders, neurological diseases, and cancer.

Depending on what is wrong, scientists can do one of several things in gene therapy:

To insert new genes directly into cells, scientists use a vehicle called a vector. Vectors are genetically engineered to deliver the necessary genes for treating the disease.

Vectors need to be able to efficiently deliver genetic material into cells, and there are different kinds of vectors. Viruses are currently the most commonly used vectors in gene therapies because they have a natural ability to deliver genetic material into cells. Before a virus can be used to carry therapeutic genes into human cells, it is modified to remove its ability to cause infectious disease.

Gene therapy can be used to modify cells inside or outside the body.When a gene therapy is used to modify cells inside the body, a doctor will inject the vector carrying the gene directly into the patient.

When gene therapy is used to modify cells outside the body, doctors take blood, bone marrow, or another tissue, and separate out specific cell types in the lab. The vector containing the desired gene is introduced into these cells. The cells are later injected into the patient, where the new gene is used to produce the desired effect.

Before a gene therapy can be marketed for use in humans, the product must be tested in clinical studies for safety and effectiveness so FDA scientists can consider whether the risks of the therapy are acceptable considering the potential benefits.

The scientific field for gene therapy products is fast-paced and rapidly evolving ushering in a new approach to the treatment of vision loss, cancer, and other serious and rare diseases. As scientists continue to make great strides in this therapy, the FDA is committed to helping speed up development by interacting with those developing products and through prompt review of groundbreaking treatments that have the potential to save lives.

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How Gene Therapy Can Cure or Treat Diseases | FDA

India can lead the One Earth, One Health vision with holistic policy-making environment: Mansukh Manda..  ETHealthWorld

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India can lead the One Earth, One Health vision with holistic policy-making environment: Mansukh Manda.. - ETHealthWorld

By: Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research

The U.S. Food and Drug Administration plays a vital role in facilitating the development and availability of innovative medical products. Products such as cellular-derived therapies, including stem cell-based products, offer the potential to treat or even cure diseases or conditions for which few effective treatment options exist.

The FDAs November 2017 regenerative medicine policy framework was developed to help facilitate and support innovation in the area of regenerative medicine therapies. As part of this framework, we encourage sponsors to take advantage of ongoing expedited programs that might be available to them, including Regenerative Medicine Advanced Therapy, breakthrough therapy, and fast track designations, to support product development and licensure.

The framework also outlines the agencys intent to exercise enforcement discretion with respect to the FDAs investigational new drug (IND) and premarket approval requirements for certain regenerative medicine products until November 2020, which was later extended through May 2021. This compliance and enforcement discretion policy gives manufacturers time to determine if certain requirements apply to their products, and if an application is needed, to prepare and submit the appropriate application to the FDA.

We are now reaffirming the timing of the end of the compliance and enforcement discretion policy for certain human cell, tissue, and cellular and tissue-based products (HCT/Ps), including regenerative medicine therapies. The period during which the FDA intends to exercise enforcement discretion with respect to the IND and premarket approval requirements for certain HCT/Ps ends on May 31, 2021, and will not be extended further.

Since November 2017, the FDA has worked with product developers to help them determine if they need to submit an IND or marketing application and, if so, how they should submit their application to the FDA. The FDA developed programs that provide opportunities for engagement between HCT/P manufacturers and the agency, including the Tissue Reference Group (TRG) Rapid Inquiry Program (TRIP). TRIP helped manufacturers of HCT/Ps, including stakeholders that market HCT/Ps to physicians or patients, obtain a rapid, preliminary, informal, non-binding assessment from the FDA regarding how specific HCT/Ps are regulated. TRIP was a temporary program of the TRG. The TRIP began in June 2019 and was extended twice. It recently ended on March 31, 2021.

Despite all of the FDAs efforts to engage industry, there continues to be broad marketing of these unapproved products for the treatment or cure of a wide range of diseases or medical conditions. Many of these unapproved products appear to be HCT/Ps that are regulated as drugs, devices and/or biological products subject to premarket approval requirements. The wide extent of the marketing of such unapproved products is evidenced by their inappropriate advertisement in various media and by the number of consumer complaints about them submitted to the FDA.

These regenerative medicine products are not without risk and are often marketed by clinics as being safe and effective for the treatment of a wide range of diseases or conditions, even though they havent been adequately studied in clinical trials. Weve said previously and want to reiterate here there is no room for manufacturers, clinics, or health care practitioners to place patients at risk through products that violate the law, including by not having an IND in effect or an approved biologics license. We will continue to take action regarding unlawfully marketed products. Our oversight of cellular and related products has included taking compliance actions, including numerous warning and untitled letters, and pursuing enforcement action for serious violations of the law.

Since December 2019, the agency has issued more than 350 letters to manufacturers, clinics, and health care providers, noting that it has come to our attention that they may be offering unapproved regenerative medicine products and reiterating the FDAs compliance and enforcement policy.

We encourage the public and patients who are considering treatment with regenerative medicine products to work with their health care providers to learn about the treatment being offered. Ask questions and understand the potential risks of treatment with unapproved products. It is critical to only seek treatment using legally marketed products, or, for unapproved products, to enroll in clinical trials under FDA oversight. The public can visit the FDAs website to find out if a particular regenerative medicine product is approved.

The FDA remains committed to helping advance the development of safe and effective regenerative medicine products, including stem cell-based products, to benefit individuals in need. We look forward to working with those who share this goal.

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Advancing Safe and Effective Regenerative Medicine Products

Active Wound Care Market Rising demand for Skin Substitutes to boost the industry (2023-2033) | CAGR of 5.5%  EIN News

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Active Wound Care Market Rising demand for Skin Substitutes to boost the industry (2023-2033) | CAGR of 5.5% - EIN News

Veterinary Orthopedic Implants Market is estimated to be 421.3 Million by 2029 with a CAGR of 5.3% - By PMI  EIN News

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Veterinary Orthopedic Implants Market is estimated to be 421.3 Million by 2029 with a CAGR of 5.3% - By PMI - EIN News