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

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 t...

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Publicat a:EMBO Mol Med
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: John Wiley and Sons Inc. 2017
Matèries:
Research Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5801507/
https://ncbi.nlm.nih.gov/pubmed/29246969
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201707988
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