Article
Allele-specific silencing therapy for Dynamin 2-related dominant centronuclear myopathy.
EMBO molecular medicine - 1 Feb 2018
Trochet Delphine, Prudhon Bernard, Beuvin Maud, Peccate Cécile, Lorain Stéphanie, Julien Laura, Benkhelifa-Ziyyat Sofia, Rabai Aymen, Mamchaoui Kamel, Ferry Arnaud, Laporte Jocelyn, Guicheney Pascale, Vassilopoulos Stéphane, Bitoun Marc
Abstract excerpt
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 autosomal-dominant centronuclear myopathy (CNM), a rare neuromuscular disorder without available treatment due to heterozygous mutations in the DNM2 gene encoding Dynamin 2. Allele-specific siRNA sequences were developed in...
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