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Effective Limb Transduction and Phenotypic Correction after Injection of rAAV8-U7 snRNA in GRMD Dogs

Archive ouverte | Le Guiner, Caroline | CCSD

In Duchenne Muscular Dystrophy (DMD) the selective removal by exon skipping of exons anking an out-of frame mutation in the dystrophin messenger can result in in-frame mRNA transcripts that are translated into shorter but functio...

Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients

Archive ouverte | Le Guiner, Caroline | CCSD

International audience. Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by mutations in the dystrophin gene, without curative treatment yet available. Our study provides, for the first t...

Adeno-Associated Virus Vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne Muscular Dystrophy (DMD)

Archive ouverte | Le Guiner, Caroline | CCSD

Duchenne Muscular Dystrophy (DMD) is a X-linked inherited muscle-wasting disease primarily affecting young boys with a prevalence of 1:5,000. The disease is caused by loss-of-function mutations in the gene encoding for the Dystrop...

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