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Muscular Dystrophy
RNA approach may do what "conventional" gene therapy can't
July 24th, 2003
A new approach effectively patches up a genetic defect in a mouse model of Duchenne muscular dystrophy, a disease that has proved 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 2003 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...
Source: Gene Therapy Weekly (2003-07-24)
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