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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Detalles Bibliográficos
Autores principales: Wang, Yiran, Xu, Chongchong, Ma, Lin, Mou, Yongchao, Zhang, Bowen, Zhou, Shanshan, Tian, Yue, Trinh, Jessica, Zhang, Xiaoqing, Li, Xue-Jun
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Life Science Alliance LLC 2019
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6435041/
https://www.ncbi.nlm.nih.gov/pubmed/30910806
http://dx.doi.org/10.26508/lsa.201800268

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6435041/
https://www.ncbi.nlm.nih.gov/pubmed/30910806
http://dx.doi.org/10.26508/lsa.201800268

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