CRISPR Helps Get Closer to Cure for Duchenne Muscular Dystrophy

Many experiments on mice have shown the promising results. There has already been successful in overcoming the symptoms of muscular dystrophy. For genetic therapy to be successful, it is not essential that defective gene is edited in all the muscular cells, instead editing a small percentage of muscular cells would be enough.

In Duchenne muscular dystrophy, the mutation causes a deficit of protein called dystrophin, resulting in the progressive loss of muscle cells. Thus if the mutated gene is edited even in the small number of muscle fibers, they can grow and take the place of defective muscle fibers.

To minimize the side effects, and improve the effectiveness of any therapy (including traditional pharmacological treatment), it is essential to find out the practical ways of targeted delivery, to make sure that therapeutic agents get delivered only to the target organs and other organs are spared.

It is where the innovation is taking place. The scientists have created a complex of gold nanoparticles and CRISPR components that when injected locally in musculature, carry out the editing process in muscles, but effects on other tissues are minimal(3).