Spinal muscular atrophy: from tissue specificity to therapeutic strategies

Spinal muscular atrophy (SMA) is the most frequent genetic cause of death in infants and toddlers. All cases of spinal muscular atrophy result from reductions in levels of the survival motor neuron (SMN) protein, and so SMN upregulation is a focus of many preclinical and clinical studies. We examine...

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Detalles Bibliográficos
Autores principales: Iascone, Daniel M., Henderson, Christopher E., Lee, Justin C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Faculty of 1000 Ltd 2015
Materias:
Review Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4311279/
https://www.ncbi.nlm.nih.gov/pubmed/25705387
http://dx.doi.org/10.12703/P7-04

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