A new approach effectively patches up a genetic defect in a mouse model of Duchenne muscular dystrophy, a disease that has proven recalcitrant to new therapies. The gene that underlies this disorder encodes a muscle protein called dystrophin. But the exceptionally large size of the dystrophin gene, and the difficulty in delivering the gene to the appropriate muscle fibers, has hindered efforts at gene therapy.
In the August issue of Nature Medicine, Terence Partridge and colleagues side-step such barriers in a mouse model. Instead of introducing a corrected form of the gene, the investigators injected a small ‘antisense’ RNA molecule into muscle. This RNA molecule binds to specific sequences in the dystrophin RNA and interferes with a process called splicing. This resulted in the production of a nearly normal dystrophin protein and, even more dramatically, improved muscle function. A single injection in mice resulted in expression of the corrected dystrophin for more than 3 months. But before testing the technique in humans, the investigators note that improvements over the current method of frequent injections -- such as delivery of RNA to the muscles via the bloodstream -- should be investigated. The new research offers hope for the approximately 1 in 3,500 children, mostly boys, born each year with the disorder.
(MRC Clinical Science Center, Hammersmith Hospital, London, UK)
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(C) Nature Medicine press release.
Message posted by: Trevor M. D'Souza
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