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

Beyret, Ergin; Liao, Hsin-Kai; Yamamoto, Mako; Hernandez-Benitez, Reyna; Fu, Yunpeng; Erikson, Galina; Reddy, Pradeep; Belmonte, Juan Carlos Izpisua

NATURE MEDICINE
2019
VL / 25 - BP / 419 - EP / +
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.

AccesS level

Green accepted

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