Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease

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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Bibliographic Details
Main Authors: Valentina Sardone, Haiyan Zhou, Francesco Muntoni, Alessandra Ferlini, Maria Sofia Falzarano
Format: Article
Language:English
Published: MDPI AG 2017-04-01
Series:Molecules
Subjects:
Duchenne Muscular Dystrophy
Spinal Muscular Atrophy
antisense oligonucleotides
clinical trials
oligonucleotides delivery
Online Access:http://www.mdpi.com/1420-3049/22/4/563

Internet

http://www.mdpi.com/1420-3049/22/4/563

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