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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Detalhes bibliográficos
Publicado no:Nature
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2021
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Article
Acesso em linha: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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