Genome Editing-Enabled HTS Assays Expand Drug Target Pathways for Charcot–Marie–Tooth Disease

[Image: see text] Copy number variation resulting in excess PMP22 protein causes the peripheral neuropathy Charcot–Marie–Tooth disease, type 1A. To broadly interrogate chemically sensitive transcriptional pathways controlling PMP22 protein levels, we used the targeting precision of TALEN-mediated ge...

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Detalhes bibliográficos
Publicado no:ACS Chem Biol
Main Authors: Inglese, James, Dranchak, Patricia, Moran, John J., Jang, Sung-Wook, Srinivasan, Rajini, Santiago, Yolanda, Zhang, Lei, Guha, Rajarshi, Martinez, Natalia, MacArthur, Ryan, Cost, Gregory J., Svaren, John
Formato: Artigo
Idioma:Inglês
Publicado em: American Chemical Society 2014
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4245164/
https://ncbi.nlm.nih.gov/pubmed/25188731
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/cb5005492
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