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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: American Chemical Society 2014
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4245164/
https://www.ncbi.nlm.nih.gov/pubmed/25188731
http://dx.doi.org/10.1021/cb5005492

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