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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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Detalles Bibliográficos
Autores principales: Wee, Claribel D., Havens, Mallory A., Jodelka, Francine M., Hastings, Michelle L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4266657/
https://www.ncbi.nlm.nih.gov/pubmed/25506695
http://dx.doi.org/10.1371/journal.pone.0115205