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Archive for the ‘Gene therapy’ Category

Was Darwin a Punk? A Q&A with Punker-Paleontologist Greg Graffin

Monday, October 11th, 2010

Editor's Note: This is an expanded version of the Q&A that will appear in the November 2010 issue of Scientific American.

Name: Greg Graffin [More]

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Greg Graffin - BadReligion - Punk rock - Punk - Shopping

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Stem Cells from Reprogrammed Adult Cells Found to Bring Along Genetic Defects of Their Donors

Monday, October 11th, 2010

Realistic stem cell therapies to replace diseased or damaged tissue may still be years away, but researchers have uncovered a promising new use for these undifferentiated cells: they can be programmed to become patient-specific laboratory models of inherited liver disease. These new tools could be useful for teasing out disease mechanisms and testing new drug therapies.

Scientists from the University of Cambridge's Institute for Medical Research obtained skin cells from 10 patients--seven who had various forms of inherited liver disease, and three healthy controls. They reprogrammed the skin cells, rejuvenating them into an embryolike state (using the four-gene approach described in 2007). The researchers then cultured these so-called induced pluripotent stem cells (iPS cells) in a mixture of chemical factors that triggered their conversion into liver cells, which had the appearance and functional properties of native liver cells.

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Stem cell - Cambridge University - Liver - Medical Research - Disease

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Parkinsonian Power Failure: Neuron Degeneration May Be Caused by a Cellular Energy System Breakdown

Monday, October 11th, 2010

In the past researchers have observed an association between poor mitochondrial function and Parkinson's disease, a neurodegenerative disorder of the central nervous system that impairs speech and motor functions and affects five million people worldwide. A new meta-analysis suggests that low expression levels of 10 related gene sets responsible for mitochondrial machinery play an important role in this disorder--all previously unlinked to Parkinson's. The study, published online today in Science Translational Medicine , further points to a master switch for these gene sets as a potential target of future therapies. [More]

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Was Darwin a Punk? A Q&A with Punker-Paleontologist Greg Graffin

Monday, October 11th, 2010

Editor's Note: This is an expanded version of the Q&A that will appear in the November 2010 issue of Scientific American.

Name: Greg Graffin [More]

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Greg Graffin - BadReligion - Punk rock - Punk - Shopping

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Stem Cells from Reprogrammed Adult Cells Found to Bring Along Genetic Defects of Their Donors

Monday, October 11th, 2010

Realistic stem cell therapies to replace diseased or damaged tissue may still be years away, but researchers have uncovered a promising new use for these undifferentiated cells: they can be programmed to become patient-specific laboratory models of inherited liver disease. These new tools could be useful for teasing out disease mechanisms and testing new drug therapies.

Scientists from the University of Cambridge's Institute for Medical Research obtained skin cells from 10 patients--seven who had various forms of inherited liver disease, and three healthy controls. They reprogrammed the skin cells, rejuvenating them into an embryolike state (using the four-gene approach described in 2007). The researchers then cultured these so-called induced pluripotent stem cells (iPS cells) in a mixture of chemical factors that triggered their conversion into liver cells, which had the appearance and functional properties of native liver cells.

[More]

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Stem cell - Cambridge University - Liver - Medical Research - Disease

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2 Genes Linked to Embryonic Brain Impairment in Down’s Syndrome

Thursday, July 22nd, 2010

Down's syndrome (DS) is an incurable, heritable disorder affecting an estimated 400,000 people in the U.S. It is characterized by impaired cognitive ability and abnormal physical growth. Whereas scientists have long known that DS is caused by inheriting an extra copy of all or part of chromosome 21 , the underlying cause of the brain defects common in Down's patients has not been fully gleaned.

Now, a collaborative team of scientists working with a mouse model of DS has discovered that just two genes are responsible for the majority of the brain abnormalities present in their animals. The scientists hope that their findings will help scientists understand brain defects in humans with the disorder as well as aid in the development of drugs to treat the cognitive impairment in Down's patients.

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Genetic disorder - Health - Conditions and Diseases - Gene - Brain

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2 Genes Linked to Embryonic Brain Impairment in Down’s Syndrome

Thursday, July 22nd, 2010

Down's syndrome (DS) is an incurable, heritable disorder affecting an estimated 400,000 people in the U.S. It is characterized by impaired cognitive ability and abnormal physical growth. Whereas scientists have long known that DS is caused by inheriting an extra copy of all or part of chromosome 21 , the underlying cause of the brain defects common in Down's patients has not been fully gleaned.

Now, a collaborative team of scientists working with a mouse model of DS has discovered that just two genes are responsible for the majority of the brain abnormalities present in their animals. The scientists hope that their findings will help scientists understand brain defects in humans with the disorder as well as aid in the development of drugs to treat the cognitive impairment in Down's patients.

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Genetic disorder - Health - Conditions and Diseases - Gene - Brain

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DNA Drugs Come of Age (preview)

Friday, July 16th, 2010

In a head-to-head competition held 10 years ago, scientists at the National Institutes of Health tested two promising new types of vaccine to see which might offer the strongest protection against one of the deadliest viruses on earth, the human immunodeficiency virus (HIV) that causes AIDS. One vaccine consisted of DNA rings called plasmids, each carrying a gene for one of five HIV proteins. Its goal was to get the recipient’s own cells to make the viral proteins in the hope they would provoke protective reactions by immune cells. Instead of plasmids, the second vaccine used another virus called an adenovirus as a carrier for a single HIV gene encoding a viral protein. The rationale for this combination was to employ a “safe” virus to catch the attention of immune cells while getting them to direct their responses against the HIV protein.

One of us (Weiner) had already been working on DNA vaccines for eight years and was hoping for a major demonstration of the plasmids’ ability to induce immunity against a dreaded pathogen. Instead the test results dealt a major blow to believers in this first generation of DNA vaccines. The DNA recipients displayed only weak immune responses to the five HIV proteins or no response at all, whereas recipients of the adenovirus-based vaccine had robust reactions. To academic and pharmaceutical company researchers, adenoviruses clearly looked like the stronger candidates to take forward in developing HIV vaccines.

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Immune system - National Institutes of Health - Vaccine - HIV - DNA

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DNA Drugs Come of Age (preview)

Friday, July 16th, 2010

In a head-to-head competition held 10 years ago, scientists at the National Institutes of Health tested two promising new types of vaccine to see which might offer the strongest protection against one of the deadliest viruses on earth, the human immunodeficiency virus (HIV) that causes AIDS. One vaccine consisted of DNA rings called plasmids, each carrying a gene for one of five HIV proteins. Its goal was to get the recipient’s own cells to make the viral proteins in the hope they would provoke protective reactions by immune cells. Instead of plasmids, the second vaccine used another virus called an adenovirus as a carrier for a single HIV gene encoding a viral protein. The rationale for this combination was to employ a “safe” virus to catch the attention of immune cells while getting them to direct their responses against the HIV protein.

One of us (Weiner) had already been working on DNA vaccines for eight years and was hoping for a major demonstration of the plasmids’ ability to induce immunity against a dreaded pathogen. Instead the test results dealt a major blow to believers in this first generation of DNA vaccines. The DNA recipients displayed only weak immune responses to the five HIV proteins or no response at all, whereas recipients of the adenovirus-based vaccine had robust reactions. To academic and pharmaceutical company researchers, adenoviruses clearly looked like the stronger candidates to take forward in developing HIV vaccines.

[More]

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Immune system - National Institutes of Health - Vaccine - HIV - DNA

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Hair Trigger: How a Cell’s Primary Cilium Functions as a Molecular Antenna

Wednesday, June 30th, 2010

It turns out that not all the hairlike cilia projecting from the surfaces of many cells in the human body are equal--there are the myriad ones for sweeping, swimming and other functions, and then there is the until recently mysterious primary cilium.

Nearly all human cells contain these numerous microscopic projections. The more abundant variety of cilia are motile; they act like oars, paddling in coordinated waves to help propel cells through fluid, or to sweep material across cellular surfaces (as in the respiratory system, where millions of cilia lining the airways help to expel mucus, dead cells and other bodily debris). By contrast, cells also contain a single, nonmotile cilium known as the primary cilium. Its presence on cells has been known for more than a century, but many believed it was a functionless evolutionary remnant.

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Cell - Biology - Cell biology - Cilium - Human body

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Hair Trigger: How a Cell’s Primary Cilium Functions as a Molecular Antenna

Wednesday, June 30th, 2010

It turns out that not all the hairlike cilia projecting from the surfaces of many cells in the human body are equal--there are the myriad ones for sweeping, swimming and other functions, and then there is the until recently mysterious primary cilium.

Nearly all human cells contain these numerous microscopic projections. The more abundant variety of cilia are motile; they act like oars, paddling in coordinated waves to help propel cells through fluid, or to sweep material across cellular surfaces (as in the respiratory system, where millions of cilia lining the airways help to expel mucus, dead cells and other bodily debris). By contrast, cells also contain a single, nonmotile cilium known as the primary cilium. Its presence on cells has been known for more than a century, but many believed it was a functionless evolutionary remnant.

[More]

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Cell - Biology - Cell biology - Cilium - Human body

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A genome story: 10th anniversary commentary by Francis Collins

Tuesday, June 29th, 2010

For those of you who like stories with simple plots and tidy endings, I must confess the tale of the Human Genome Project isn't one of those. The story didn't reach its conclusion when we unveiled the first draft of the human genetic blueprint at the White House on June 26, 2000. Nor did it end on April 14, 2003, with the completion of a finished, reference sequence. [More]

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Human Genome Project - White House - Biology - genetic - Francis Collins

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A genome story: 10th anniversary commentary by Francis Collins

Tuesday, June 29th, 2010

For those of you who like stories with simple plots and tidy endings, I must confess the tale of the Human Genome Project isn't one of those. The story didn't reach its conclusion when we unveiled the first draft of the human genetic blueprint at the White House on June 26, 2000. Nor did it end on April 14, 2003, with the completion of a finished, reference sequence. [More]

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Human Genome Project - White House - Biology - genetic - Francis Collins

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Vaccines Derived from Patients’ Tumor Cells Are Individualizing Cancer Treatment

Saturday, June 26th, 2010

The first discovery of a cancer gene marker--the BRAF oncogene for melanoma and colorectal malignancies--back in 2002 changed the way many researchers thought about cancer treatment. Rather than approach the disease based on what region of the body it stemmed from, scientists began to identify cancers in terms of their genetic signatures. Researchers now recognize more than 200 kinds of cancer--all genetically unique. [More]

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Cancer - Health - Conditions and Diseases - Colorectal cancer - Management of cancer

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Vaccines Derived from Patients’ Tumor Cells Are Individualizing Cancer Treatment

Saturday, June 26th, 2010

The first discovery of a cancer gene marker--the BRAF oncogene for melanoma and colorectal malignancies--back in 2002 changed the way many researchers thought about cancer treatment. Rather than approach the disease based on what region of the body it stemmed from, scientists began to identify cancers in terms of their genetic signatures. Researchers now recognize more than 200 kinds of cancer--all genetically unique. [More]

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Cancer - Health - Conditions and Diseases - Colorectal cancer - Management of cancer

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Cancer Therapy Goes Viral: Progress Is Made Tackling Tumors with Viruses

Thursday, June 24th, 2010

The adapted virus that immunized hundreds of millions of people against smallpox has now been enlisted in the war on cancer. Vaccinia poxvirus joins a herpesvirus and a host of other pathogens on a growing list of engineered viruses entering late-stage human testing against cancer. [More]

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Cancer - Smallpox - Virus - Vaccinia - Health

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Cancer Therapy Goes Viral: Progress Is Made Tackling Tumors with Viruses

Thursday, June 24th, 2010

The adapted virus that immunized hundreds of millions of people against smallpox has now been enlisted in the war on cancer. Vaccinia poxvirus joins a herpesvirus and a host of other pathogens on a growing list of engineered viruses entering late-stage human testing against cancer. [More]

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Cancer - Smallpox - Virus - Vaccinia - Health

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Large-Scale Autism Study Reveals Disorder’s Genetic Complexity

Saturday, June 12th, 2010

The vast array of behaviors that are seen in autism spectrum disorder seems to cover an even deeper range of genetic complexity just below the surface. And the largest genetic study of autistic children and their parents to date has located a host of new variations in autistic individuals. [More]

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Autism spectrum - Autism - Health - Mental health - Disorders

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Large-Scale Autism Study Reveals Disorder’s Genetic Complexity

Saturday, June 12th, 2010

The vast array of behaviors that are seen in autism spectrum disorder seems to cover an even deeper range of genetic complexity just below the surface. And the largest genetic study of autistic children and their parents to date has located a host of new variations in autistic individuals. [More]

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Autism spectrum - Autism - Health - Mental health - Disorders

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Alzheimer’s: Forestalling the Darkness with New Approaches (preview)

Friday, May 28th, 2010

In his magical-realist masterpiece One Hundred Years of Solitude , Colombian author Gabriel García Márquez takes the reader to the mythical jungle village of Macondo, where, in one oft-recounted scene, residents suffer from a disease that causes them to lose all memory. The malady erases “the name and notion of things and finally the identity of people.” The symptoms persist until a traveling gypsy turns up with a drink “of a gentle color” that returns them to health.

In a 21st-century parallel to the townspeople of Macondo, a few hundred residents from Medellín, Colombia, and nearby coffee-growing areas may get a chance to assist in the search for something akin to a real-life version of the gypsy’s concoction. Medellín and its environs are home to the world’s largest contingent of individuals with a hereditary form of Alzheimer’s disease. Members of 25 extended families, with 5,000 members, develop early-onset Alzheimer’s, usually before the age of 50, if they harbor an aberrant version of a particular gene.

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Alzheimer - Macondo - One Hundred Years of Solitude - Health - Conditions and Diseases

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