Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy

Published 2011 View Full Article

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Title
Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy
Authors
Keywords
-
Journal
MOLECULAR THERAPY
Volume 20, Issue 1, Pages 119-126
Publisher
Elsevier BV
Online
2011-10-27
DOI
10.1038/mt.2011.232

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