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Nat Med. 2019 Mar;25(3):419-422. doi: 10.1038/s41591-019-0343-4. Epub 2019 Feb 18.

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

Author information

1
Salk Institute for Biological Studies, La Jolla, CA, USA.
2
Universidad Católica San Antonio de Murcia, Murcia, Spain.
3
Salk Institute for Biological Studies, La Jolla, CA, USA. belmonte@salk.edu.

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.

PMID:
30778240
DOI:
10.1038/s41591-019-0343-4
[Indexed for MEDLINE]

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