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Benefits of therapy by Dynamin 2 mutant specific silencing are maintained with time in a mouse model of dominant centronuclear myopathy

Archive ouverte | Trochet, Delphine | CCSD

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Benefits of therapy by dynamin-2-mutant-specific silencing are maintained with time in a mouse model of dominant centronuclear myopathy

Archive ouverte | Trochet, Delphine | CCSD

International audience. Dominant dynamin 2 (DNM2) mutations are responsible for the autosomal dominant centronuclear myopathy (AD-CNM), a rare progressive neuromuscular disorder ranging from severe neonatal to mild ...

Allele-specific silencing therapy for Dynamin 2-related dominant centronuclear myopathy

Archive ouverte | Trochet, Delphine | CCSD

International audience. Rapid advances in allele-specific silencing by RNA interference established a strategy of choice to cure dominant inherited diseases by targeting mutant alleles. We used this strategy for aut...

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