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

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

Abstract 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 treatm...

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Main Authors: Delphine Trochet, Bernard Prudhon, Maud Beuvin, Cécile Peccate, Stéphanie Lorain, Laura Julien, Sofia Benkhelifa‐Ziyyat, Aymen Rabai, Kamel Mamchaoui, Arnaud Ferry, Jocelyn Laporte, Pascale Guicheney, Stéphane Vassilopoulos, Marc Bitoun
Format: Article
Language:English
Published: Wiley 2018-02-01
Series:EMBO Molecular Medicine
Subjects:
allele‐specific silencing therapy
centronuclear myopathy
Dynamin 2
RNA interference
Online Access:https://doi.org/10.15252/emmm.201707988

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