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Targeting SR Proteins Improves SMN Expression in Spinal Muscular Atrophy Cells

Spinal muscular atrophy (SMA) is one of the most common inherited causes of pediatric mortality. SMA is caused by deletions or mutations in the survival of motor neuron 1 (SMN1) gene, which results in SMN protein deficiency. Humans have a centromeric copy of the survival of motor neuron gene, SMN2,...

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書目詳細資料
發表在:PLoS One
Main Authors: Wee, Claribel D., Havens, Mallory A., Jodelka, Francine M., Hastings, Michelle L.
格式: Artigo
語言:Inglês
出版: Public Library of Science 2014
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC4266657/
https://ncbi.nlm.nih.gov/pubmed/25506695
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0115205
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