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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Vydáno v:Nucleic Acids Res
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2020
Témata:
Gene regulation, Chromatin and Epigenetics
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7145652/
https://ncbi.nlm.nih.gov/pubmed/31713617
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkz1070
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