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Targeted PMP22 TATA-box editing by CRISPR/Cas9 reduces demyelinating neuropathy of Charcot-Marie-Tooth disease type 1A in mice

Charcot-Marie-Tooth 1A (CMT1A) is the most common inherited neuropathy without a known therapy, which is caused by a 1.4 Mb duplication on human chromosome 17, which includes the gene encoding the peripheral myelin protein of 22 kDa (PMP22). Overexpressed PMP22 protein from its gene duplication is t...

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Detalles Bibliográficos
Autores principales: Lee, Ji-Su, Lee, Jae Y, Song, Dong W, Bae, Hee S, Doo, Hyun M, Yu, Ho S, Lee, Kyu J, Kim, Hee K, Hwang, Hyun, Kwak, Geon, Kim, Daesik, Kim, Seokjoong, Hong, Young B, Lee, Jung M, Choi, Byung-Ok
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7145652/
https://www.ncbi.nlm.nih.gov/pubmed/31713617
http://dx.doi.org/10.1093/nar/gkz1070