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Identification of Novel Compounds That Increase SMN Protein Levels Using an Improved SMN2 Reporter Cell Assay

Spinal muscular atrophy (SMA) is a neurodegenerative disorder that is characterized by progressive loss of motor neuron function. It is caused by the homozygous loss of the SMN1 (survival of motor neuron 1) gene and a decrease in full-length SMN protein. SMN2 is a nearly identical homolog of SMN1 th...

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Detalhes bibliográficos
Main Authors: Cherry, Jonathan J., Evans, Matthew C., Ni, Jake, Cuny, Gregory D., Glicksman, Marcie A., Androphy, Elliot J.
Formato: Artigo
Idioma:Inglês
Publicado em: 2012
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3870337/
https://ncbi.nlm.nih.gov/pubmed/22233647
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1087057111431605
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