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

被引：112

作者：

Beyret, Ergin ^{[1
]}

Liao, Hsin-Kai ^{[1
]}

Yamamoto, Mako ^{[1
,2
]}

Hernandez-Benitez, Reyna ^{[1
]}

Fu, Yunpeng ^{[1
]}

Erikson, Galina ^{[1
]}

Reddy, Pradeep ^{[1
]}

Belmonte, Juan Carlos Izpisua ^{[1
]}

机构：

[1] Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA

[2] Univ Catolica San Antonio Murcia, Murcia, Spain

来源：

NATURE MEDICINE | 2019年 / 25卷 / 03期

关键词：

PREMATURE; LAMINOPATHIES; PRELAMIN;

D O I：

10.1038/s41591-019-0343-4

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

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.

引用

页码：419 / +

页数：11

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