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Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Archive ouverte | Le Guiner, Caroline, C. | CCSD

International audience. Duchenne muscular dystrophy (DMD) is an incurable X-linked muscle-wasting disease caused by mutations in the dystrophin gene. Gene therapy using highly functional microdystrophin genes and re...

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