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

Ergin Beyret | Zendy; HsinKai Liao | Zendy; Mako Yamamoto | Zendy; Reyna HernándezBenítez | Zendy; Yunpeng Fu | Zendy; Galina Erikson | Zendy; Pradeep Reddy | Zendy; Juan Carlos Izpisúa Belmonte | Zendy

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

Author(s) -

Ergin Beyret,

HsinKai Liao,

Mako Yamamoto,

Reyna HernándezBenítez,

Yunpeng Fu,

Galina Erikson,

Pradeep Reddy,

Juan Carlos Izpisúa Belmonte

Publication year - 2019

Publication title -

nature medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.536

H-Index - 547

eISSN - 1546-170X

pISSN - 1078-8956

DOI - 10.1038/s41591-019-0343-4

Subject(s) - progeria , lmna , lamin , crispr , biology , genetics , mutation , cancer research , gene

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.

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