Benefits of therapy by dynamin-2-mutant-specific silencing are maintained with time in a mouse model of dominant centronuclear myopathy
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Molecular Medicine
Reference48 articles.
1. Centronuclear myopathies: a widening concept;Romero;Neuromuscul. Disord.,2010
2. Mutation spectrum in the large gtpase dynamin 2, and genotype-phenotype correlation in autosomal dominant centronuclear myopathy;Böhm;Hum. Mutat.,2012
3. Mutations in dynamin 2 cause dominant centronuclear myopathy;Bitoun;Nat. Genet.,2005
4. Some DNM2 mutations cause extremely severe congenital myopathy and phenocopy myotubular myopathy;Biancalana;Acta Neuropathol. Commun.,2018
5. A novel dynamin-2 gene mutation associated with a late-onset centronuclear myopathy with necklace fibres;Casar-Borota;Neuromuscul. Disord.,2015
Cited by 2 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献
1. Optimized allele-specific silencing of the dominant-negative COL6A1 G293R substitution causing collagen VI-related dystrophy;Molecular Therapy - Nucleic Acids;2024-06
2. La myopathie centronucléaire liée au gène de la dynamine 2;médecine/sciences;2023-11
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