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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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Dades bibliogràfiques
Publicat a:ACS Chem Biol
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: American Chemical Society 2014
Accés en línia: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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