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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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Detalles Bibliográficos
Autores principales: Sardone, Valentina, Zhou, Haiyan, Muntoni, Francesco, Ferlini, Alessandra, Falzarano, Maria Sofia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6154734/
https://www.ncbi.nlm.nih.gov/pubmed/28379182
http://dx.doi.org/10.3390/molecules22040563