Category Archives: Gene therapy

A decade ago biologists and nonbiologists alike gushed with optimism about the medical promise of the $3-billion Human Genome Project. In announcing the first rough draft of the human “book of life” at a White House ceremony in the summer of 2000, President Bill Clinton predicted that the genome project would “revolutionize the diagnosis, prevention and treatment of most, if not all, human diseases.”

A year earlier Francis S. Collins, then head of the National Human Genome Research Institute and perhaps the project’s most tireless enthusiast, painted a grand vision of the “personalized medicine” likely to emerge from the project by the year 2010: genetic tests indicating a person’s risk for heart disease, cancer and other common maladies would be available, soon to be followed by preventives and therapies tailored to the individual.

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When Jim Laidler’s oldest son, Benjamin, was diagnosed with autism, he and his wife started looking for help. “The neurologists were saying, ‘We don’t know what causes autism, and we don’t know what the outcome for your son will be,’” Laidler relates. “No one was saying, ‘Here’s what causes it; here’s what treats it.’”

But when the Laidlers, who live in Portland, Ore., searched the Web, they found dozens of “biomedical” treatments that promised to improve or even cure Benjamin’s inability to talk, interact socially or control his movements. So the parents tried them on their son. They began with vitamin B6 and magnesium, the nutritional supplements dimethylglycine and tri­methylglycine, vitamin A, gluten- and casein-free diets, the digestive hormone secretin, and chelation, a drug therapy de­signed to purge the body of lead and mercury. They applied the purported treatments to Benjamin’s little brother, David, who also was diagnosed with autism. Chelation did not seem to help much. Any effect from secretin was hard to tell. The diets showed promise; the Laidlers hauled special food with them everywhere. And Mom and Dad continued to feed the boys dozens of supplements, calibrating doses up and down with every change in behavior.

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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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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]

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]

Greg Graffin - BadReligion - Punk rock - Punk - Shopping

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

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

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

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

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