A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy

Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality. Most SMA cases are associated with the low levels of SMN owing to deletion of Survival Motor Neuron 1 (SMN1). SMN2, a nearly identical copy of SMN1, fails to compensate for the loss of SMN1 due to predominant skipping of...

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Bibliographic Details
Main Authors: Singh, Natalia N., Shishimorova, Maria, Cao, Lu Cheng, Gangwani, Laxman, Singh, Ravindra N.
Format: Artigo
Language:Inglês
Published: 2009
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Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC2734876/
https://ncbi.nlm.nih.gov/pubmed/19430205
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