A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice

In the search for the most efficacious antisense oligonucleotides (AOs) aimed at inducing SMN2 exon 7 inclusion, we systematically assessed three AOs, PMO25 (−10, −34), PMO18 (−10, −27), and PMO20 (−10, −29), complementary to the SMN2 intron 7 splicing silencer (ISS-N1). PMO25 was the most efficacio...

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Main Authors: Zhou, Haiyan, Janghra, Narinder, Mitrpant, Chalermchai, Dickinson, Rachel L., Anthony, Karen, Price, Loren, Eperon, Ian C., Wilton, Stephen D., Morgan, Jennifer, Muntoni, Francesco
格式: Artigo
語言:Inglês
出版: Mary Ann Liebert, Inc. 2013
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Research Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3609631/
https://ncbi.nlm.nih.gov/pubmed/23339722
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.211
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