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General interest items edited by Janice Flahiff

[Press release] NIH researchers tackle thorny side of gene therapy

NIH researchers tackle thorny side of gene therapy 

From the 20 January 2015 press release

 

NIH researchers tackle thorny side of gene therapy

Pre-clinical studies in mice reveal ways to reduce cancer risk with modified treatment

NHGRI researchers conduct laboratory investigations to advance gene therapy. Watch the video featuring Dr. Charles Venditti and Dr. Randy Chandler: YouTube video Methylmalonic Acidemia (MMA) Gene Therapy
Lab technnician with a pipette

Bethesda, Md., Tues., Jan. 20, 2015 – National Institutes of Health researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome Research Institute (NHGRI), part of NIH, published their research in the Jan. 20, 2015, online issue of the Journal of Clinical Investigation.

“Effective and safe gene therapies have the potential to dramatically reverse diseases that are life-threatening for affected children,” said NHGRI Scientific Director Dan Kastner, M.D., Ph.D. “This study is an important step in developing gene therapies that can be safely used to benefit patients.”

Toxic side effects actually are rarely observed by researchers who have designed gene therapies using an adeno-associated virus (AAV) as a vector to deliver the corrected gene to a specific point in the cell’s DNA. AAVs are small viruses that infect humans but do not cause disease. A vector is a DNA molecule of AAV used as a vehicle to carry corrected genetic material into a cell. AAV viruses are uniquely suited for gene therapy applications.

But one prior study did find an association between AAV and the occurrence of liver cancer. The present research addresses this problem in gene therapy for an inherited disease in children called methylmalonic acidemia, or MMA. For 10 years, NHGRI researchers have worked toward a gene therapy to treat MMA. The condition affects as many as 1 in 67,000 children born in the United States. Affected children are unable to properly metabolize certain amino acids consumed in their diet, which can damage a number of organs and lead to kidney failure. MMA patients also suffer from severe metabolic instability, failure to thrive, intellectual and physical disabilities, pancreatitis, anemia, seizures, vision loss and strokes. The most common therapy is a restrictive diet, but doctors must resort to dialysis or kidney or liver transplants when the disease progresses.

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January 23, 2015 - Posted by | Medical and Health Research News | , , , , ,

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