December 2005 – Gene therapy may help muscular dys

STANFORD, Calif., Dec 27, 2005 (UPI via COMTEX) -- A gene therapy that has shown promise in skin disease and hemophilia might one day be useful for treating muscular dystrophy, says a U.S. study.

Researchers at Stanford University School of Medicine used gene therapy to introduce a healthy copy of the gene dystrophin into mice with a condition that mimics muscular dystrophy.

The dystrophin gene is mutated and as a result produces a defective protein in the roughly 20,000 people in the United States with the most common form of the disease.

Researchers have tried several different gene therapy techniques with variable success, according to Dr. Thomas Rando.

The new gene therapy technique Rando and postdoctoral fellow Carmen Bertoni used was developed by Michele Calos, associate professor of genetics.

One of the main advantages of this method is that it could potentially provide a long-term fix for a variety of genetic diseases, including muscular dystrophy, says Rando.

The study is scheduled to be published online in the Proceedings of the National Academy of Sciences the week of Jan. 2nd, 2006