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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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| Publicado no: | EMBO Mol Med |
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| Main Authors: | , , , , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
John Wiley and Sons Inc.
2017
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| Assuntos: | |
| Acesso em linha: | 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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