The lifespan of mice with Hutchinson Gilford progeria syndrome is extended by a single-dose CRISPR/Cas9 treatment
Hutchinson Gilford progeria (HGPS), a rare genetic disorder that can be fatal, is characterized by symptoms similar to accelerated aging. The main genetic cause of the syndrome is a mutation in the lamin A gene that results in the production of progerin, a toxic isoform. We show here that a single dose of CRISPR Cas9 components delivered by an adenoassociated virus suppresses HGPS using a mouse model.
Source:
https://www.nature.com/articles/s41591-019-0343-4
