Article
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.
Nature - 1 Jan 2021
Koblan Luke W, Erdos Michael R, Wilson Christopher, Cabral Wayne A, Levy Jonathan M, Xiong Zheng-Mei, Tavarez Urraca L, Davison Lindsay M, 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
Abstract excerpt
Hutchinson-Gilford progeria syndrome (HGPS or progeria) is typically caused by a dominant-negative C•G-to-T•A mutation (c.1824 C>T; p.G608G) in LMNA, the gene that encodes nuclear lamin A. This mutation causes RNA mis-splicing that produces progerin, a toxic protein that induces rapid ageing and shortens the lifespan of children with progeria to approximately 14 years1-4. Adenine base editors (ABEs) convert...
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