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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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Publicat a:Molecules
Autors principals: Sardone, Valentina, Zhou, Haiyan, Muntoni, Francesco, Ferlini, Alessandra, Falzarano, Maria Sofia
Format: Artigo
Idioma:Inglês
Publicat: MDPI 2017
Matèries:
Accés en línia: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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