Gene therapy one step closer to reality

A breakthrough gene therapy study by Italian scientists demonstrated for the first time the possibility for body-wide gene therapy for Duchenne muscular dystrophy (DMD) in mice.

DMD is a genetic disease affecting mostly boys, with symptoms of muscular weakness, difficulty in walking, running, hopping, and muscle deformities. DMD is due to mutations in the dystrophin gene, with two-thirds of the cases being hereditary. A gene therapy approach is the only way to restore correct muscule function in DMD cases, but until now, this has proved troublesome.

The major problem is getting a correctly-functioning copy of the dystrophin gene to all muscles. To reach most muscles in mice bodies, the researchers of this latest study used a modified virus to inject a functional copy of the dystrophin gene. They tracked its body-wide targeting using genetic markers and showed that the muscles regained approximately normal strength, thus showing its effectiveness.

Interestingly, not all muscle cells picked up the functional dystrophin gene copy, but they still regained strength. Even better, the results raise the hope that the virus delivery mechanism might be so effective as to create a once-in-a-lifetime treatment for DMD patients.

Applying this work to humans will take a lot of work and further studies. However, this is a solid step opening a door of hope for DMD sufferers.

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