A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular Atrophy

A Short Antisense Oligonucleotide Ameliorates Symptoms of Severe Mouse Models of Spinal Muscular Atrophy

Recent reports underscore the unparalleled potential of antisense-oligonucleotide (ASO)-based approaches to ameliorate various pathological conditions. However, in vivo studies validating the effectiveness of a short ASO (<10-mer) in the context of a human disease have not been performed. One dis...

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Bibliografiska uppgifter
Huvudupphovsmän: Jeffrey M Keil, Joonbae Seo, Matthew D Howell, Walter H Hsu, Ravindra N Singh, Christine J DiDonato
Materialtyp: Artikel
Språk:English
Publicerad: Elsevier 2014-01-01
Serie:Molecular Therapy: Nucleic Acids
Ämnen:
antisense oligonucleotide
cardiac
short ASO
skeletal muscle
SMA mouse model
SMN2
spinal muscular atrophy
splicing
Länkar:http://www.sciencedirect.com/science/article/pii/S2162253116303122

Internet

http://www.sciencedirect.com/science/article/pii/S2162253116303122

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