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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7872200/
https://www.ncbi.nlm.nih.gov/pubmed/33408413
http://dx.doi.org/10.1038/s41586-020-03086-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7872200/
https://www.ncbi.nlm.nih.gov/pubmed/33408413
http://dx.doi.org/10.1038/s41586-020-03086-7

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