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Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease

Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular Atrophy are neurodegenerative genetic diseases characterized primarily by muscle weakness and wasting. Until recently there were no effective therapies for these conditions, but antisense oligonucleotides, a new class of...

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Detalhes bibliográficos
Publicado no:Molecules
Main Authors: Sardone, Valentina, Zhou, Haiyan, Muntoni, Francesco, Ferlini, Alessandra, Falzarano, Maria Sofia
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6154734/
https://ncbi.nlm.nih.gov/pubmed/28379182
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/molecules22040563
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