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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Bibliographic Details
Main Authors: Li, Darrick K., Tisdale, Sarah, Espinoza-Derout, Jorge, Saieva, Luciano, Lotti, Francesco, Pellizzoni, Livio
Format: Artigo
Language:Inglês
Published: Public Library of Science 2013
Subjects:
Research Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3744461/
https://ncbi.nlm.nih.gov/pubmed/23967270
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0071965
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