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

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by loss of survival motor neuron-1 (SMN1). A nearly identical copy gene, SMN2, is present in all SMA patients. Although the SMN2 coding sequence has the potential to produce full-length SMN, nearly 90% of SMN2-derived transcripts ar...

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Detalhes bibliográficos
Main Authors: Osman, Erkan Y, Yen, Pei-Fen, Lorson, Christian L
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2012
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3255575/
https://ncbi.nlm.nih.gov/pubmed/22031236
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.232
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