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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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Detalhes bibliográficos
Publicado no:PLoS One
Main Authors: Wee, Claribel D., Havens, Mallory A., Jodelka, Francine M., Hastings, Michelle L.
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2014
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Acesso em linha: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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