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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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Pubblicato in:Molecules
Autori principali: Sardone, Valentina, Zhou, Haiyan, Muntoni, Francesco, Ferlini, Alessandra, Falzarano, Maria Sofia
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2017
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Accesso online: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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