NIH breakthrough in gene therapy for methylmalonic acidemia.

Researchers from the National Institutes of Health (NIH) have made significant progress in understanding the elevated cancer risk associated with gene therapy. The study was published in the Jan 20, 2015, online issue of Journal of Clinical Investigation.

“Effective and safe gene therapies have the potential to dramatically reverse diseases that are life-threatening for affected children,” said National Human Genome Research Institute (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.”

NHGRI researchers have been developing gene therapy to treat methylmalonic acidemia (MMA) for 10 years now. The disease affects 1 in 67,000 children born in the United States and can have severe effects. Although a restricted diet can be used to control the disease, dialysis or organ transplants are needed if it progresses too far.

For the full NIH statement on this very promising piece of research click here

The full study can be read here