In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice-Reference-Cited by-同舟云学术

In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice

Published:2021-01-06 Issue:7843 Volume:589 Page:608-614
ISSN:0028-0836
Container-title:Nature
language:en
Short-container-title:Nature

Author:

Koblan Luke W.^ORCID,Erdos Michael R.^ORCID,Wilson Christopher^ORCID,Cabral Wayne A.^ORCID,Levy Jonathan M.^ORCID,Xiong Zheng-Mei,Tavarez Urraca L.^ORCID,Davison Lindsay M.,Gete Yantenew G.,Mao Xiaojing,Newby Gregory A.,Doherty Sean P.,Narisu Narisu^ORCID,Sheng Quanhu,Krilow Chad,Lin Charles Y.^ORCID,Gordon Leslie B.,Cao Kan^ORCID,Collins Francis S.^ORCID,Brown Jonathan D.^ORCID,Liu David R.^ORCID

Publisher

Springer Science and Business Media LLC

Subject

Multidisciplinary

Link

http://www.nature.com/articles/s41586-020-03086-7.pdf

Reference64 articles.

1. Eriksson, M. et al. Recurrent de novo point mutations in lamin A cause Hutchinson–Gilford progeria syndrome. Nature 423, 293–298 (2003).

2. De Sandre-Giovannoli, A. et al. Lamin A truncation in Hutchinson–Gilford progeria. Science 300, 2055 (2003).

3. Gordon, L. B. et al. Impact of farnesylation inhibitors on survival in Hutchinson–Gilford progeria syndrome. Circulation 130, 27–34 (2014).

4. Gordon, L. B., Brown, W. T. & Collins, F. S. Hutchinson–Gilford Progeria Syndrome (GeneReviews, 2019).

5. Gaudelli, N. M. et al. Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage. Nature 551, 464–471 (2017).

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