Optimization of a series of heterocycles as survival motor neuron gene transcription enhancers

Spinal muscular atrophy (SMA) is a neurodegenerative disorder that results from mutations in the SMN1 gene, leading to survival motor neuron (SMN) protein deficiency. One therapeutic strategy for SMA is to identify compounds that enhance the expression of the SMN2 gene, which normally only is a mino...

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Publicado en:Bioorg Med Chem Lett
Main Authors: Choi, Sungwoon, Calder, Alyssa N., Miller, Eliza H., Anderson, Kierstyn P., Fiejtek, Dawid K., Rietz, Anne, Li, Hongxia, Cherry, Jonathan J., Quist, Kevin M., Xing, Xuechao, Glicksman, Marcie A., Cuny, Gregory D., Lorson, Christian L., Androphy, Elliot A., Hodgetts, Kevin J.
Formato: Artigo
Idioma:Inglês
Publicado: 2017
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC5701662/
https://ncbi.nlm.nih.gov/pubmed/29103974
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bmcl.2017.10.066
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