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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...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2020
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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 |
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