Drug screening with human SMN2 reporter identifies SMN protein stabilizers to correct SMA pathology

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, is caused by reduced levels of functional survival motor neuron (SMN) protein. To identify therapeutic agents for SMA, we established a versatile SMN2-GFP reporter line by targeting the human SMN2 gene. We then screened a...

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Detalhes bibliográficos
Publicado no:Life Sci Alliance
Main Authors: Wang, Yiran, Xu, Chongchong, Ma, Lin, Mou, Yongchao, Zhang, Bowen, Zhou, Shanshan, Tian, Yue, Trinh, Jessica, Zhang, Xiaoqing, Li, Xue-Jun
Formato: Artigo
Idioma:Inglês
Publicado em: Life Science Alliance LLC 2019
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6435041/
https://ncbi.nlm.nih.gov/pubmed/30910806
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.26508/lsa.201800268
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