New Gene Approach To Muscular Dystrophy – Neither Add Nor Delete, Just Edit

Duchenne Muscular Dystrophy, or DMD, the most common form of muscular dystrophy, is a genetic disorder caused by a mutation in a gene that can code for dystrophin, a protein in the muscles. This disorder is terminal, and death usually occurs before the age of 30 for the patients.

DMD, which is characterized by muscle weakness and calf enlargement, affects mostly boys. It is estimated that there are roughly 30,000 DMD patients in the U.S., EU and Japan.

Currently, there is no effective treatment for Duchenne Muscular Dystrophy. Stem cells, gene therapy or small-molecule drugs are some of the therapeutic strategies that are being tested as a treatment for DMD.

The common gene therapy approach for DMD involves adding a healthy copy of the gene to make up for the faulty gene, and there may be some unforeseen issues associated with it.

But guess what?

Duke University biomedical engineers have found a new approach to gene therapy, which they believe could be safer and more stable than the current methods.

In the new approach, the faulty gene is fixed by making use of genetic 'editing' technique, to help make it start producing a functional protein again. The novel method was tested in cell samples from Duchenne patients, and is now being tested in animal models of the disease.

by RTT Staff Writer

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New Gene Approach To Muscular Dystrophy - Neither Add Nor Delete, Just Edit