Gene Therapy Used To Assist In Treating Muscle Disorders

Gene Therapy Used To Assist In Treating Muscle Disorders

According to the researchers Thomas Rando MD, PhD, and Carmen Bertoni of Stanford University School of Medicine, the new gene therapy that has shown promise for skin diseases and hemophilia A (a genetic blood disorder) might one day be useful for treating muscle disorders like muscular dystrophy.

In the study, the researchers used gene therapy technique to insert a corrective copy of the dystrophin gene into genetically engineered 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 U.S. with the most common form of disease.

In muscular dystrophy, the muscle cells break down and are slowly superseded by adipose tissue. As a result people with this disease remained confined to wheel chair and usually die in their early twenties. There is still no treatment that can stop or reverse the progression of muscular dystrophy that is why gene therapy remains a hope for patients with muscular dystrophy.

The gene therapy technique researchers Thomas Rando and Carmen Bertoni used was developed by Michele Calos, PhD, Associate Professor of Genetics. One of the main advantages of this method is that it could potentially provide long term solution for variety of hereditary diseases including muscular dystrophy.

During the research, Bertoni used a standard gene therapy method to insert two genes-dystrophin and a gene that makes a shining protein into genetically manipulated mouse that shows symptoms of muscular dystrophy. She found that in mice producing insufficient dystrophin, she could find the shining protein slowly leak out of the cell. This leakiness indicates that the cell is not healed. In contrast, when she used Calos' gene therapy method to insert the genes, the muscle cell contained high levels of dystrophin disseminated along the length of the cell and the shining protein stayed within the cell, suggesting that the ample amount of dystrophin repaired the indisposed muscle.

Both Rando and Calos point out that this technique of gene therapy to introduce genes will pave the way for development of treatment for muscular dystrophy and other diseases such as skin diseases and hemophilia A.

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Gene Therapy Used To Assist In Treating Muscle Disorders