In Vivo Base Editing Rescues Hutchinson-Gilford Progeria Syndrome in Mice

Hutchinson-Gilford progeria syndrome (HGPS) is typically caused by a dominant-negative C•G-to-T•A mutation (c.1824 C>T, G608G) in LMNA, the nuclear lamin A gene. This mutation causes RNA mis-splicing that produces progerin, a toxic protein that induces rapid aging and shortens lifespan to ~14 yea...

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書誌詳細
出版年:Nature
主要な著者: Koblan, Luke W., Erdos, Michael R., Wilson, Christopher, Cabral, Wayne A., Levy, Jonathan M., Xiong, Zheng-Mei, Tavarez, Urraca L., Davison, Lindsay, Gete, Yantenew G., Mao, Xiaojing, Newby, Gregory A., Doherty, Sean P., Narisu, Narisu, Sheng, Quanhu, Krilow, Chad, Lin, Charles Y., Gordon, Leslie B., Cao, Kan, Collins, Francis S., Brown, Jonathan D., Liu, David R.
フォーマット: Artigo
言語:Inglês
出版事項: 2021
主題:
Article
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC7872200/
https://ncbi.nlm.nih.gov/pubmed/33408413
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41586-020-03086-7
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