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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Main Authors: Jeffrey M Keil, Joonbae Seo, Matthew D Howell, Walter H Hsu, Ravindra N Singh, Christine J DiDonato
格式: Artigo
語言:Inglês
出版: Elsevier 2014-01-01
叢編:Molecular Therapy: Nucleic Acids
主題:
antisense oligonucleotide
cardiac
short ASO
skeletal muscle
SMA mouse model
SMN2
spinal muscular atrophy
splicing
在線閱讀:http://www.sciencedirect.com/science/article/pii/S2162253116303122
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