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A Cell System for Phenotypic Screening of Modifiers of SMN2 Gene Expression and Function

Spinal muscular atrophy (SMA) is an inherited neurodegenerative disease caused by homozygous inactivation of the SMN1 gene and reduced levels of the survival motor neuron (SMN) protein. Since higher copy numbers of the nearly identical SMN2 gene reduce disease severity, to date most efforts to devel...

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Detalles Bibliográficos
Autores principales:	Li, Darrick K., Tisdale, Sarah, Espinoza-Derout, Jorge, Saieva, Luciano, Lotti, Francesco, Pellizzoni, Livio
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2013
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3744461/ https://www.ncbi.nlm.nih.gov/pubmed/23967270 http://dx.doi.org/10.1371/journal.pone.0071965

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