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Single-dose CRISPR-Cas9 therapy extends lifespan of mice with Hutchinson-Gilford progeria syndrome.

Abstract：

:Hutchinson-Gilford progeria syndrome (HGPS) is a rare lethal genetic disorder characterized by symptoms reminiscent of accelerated aging. The major underlying genetic cause is a substitution mutation in the gene coding for lamin A, causing the production of a toxic isoform called progerin. Here we show that reduction of lamin A/progerin by a single-dose systemic administration of adeno-associated virus-delivered CRISPR-Cas9 components suppresses HGPS in a mouse model.

journal_name

Nat Med

journal_title

Nature medicine

authors

Beyret E,Liao HK,Yamamoto M,Hernandez-Benitez R,Fu Y,Erikson G,Reddy P,Izpisua Belmonte JC

doi

10.1038/s41591-019-0343-4

subject

Has Abstract

pub_date

2019-03-01 00:00:00

pages

419-422

issue

3

eissn

1078-8956

issn

1546-170X

pii

10.1038/s41591-019-0343-4

journal_volume

25

pub_type

杂志文章

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