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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,...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Public Library of Science
2014
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| 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 |