Journal
NATURE MEDICINE
Volume 25, Issue 3, Pages 419-+Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/s41591-019-0343-4
Keywords
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Funding
- Catharina Foundation
- Muscular Dystrophy Association
- NIH-NCI CCSG [P30 014195]
- Helmsley Trust
- Progeria Research Foundation
- Universidad Catolica San Antonio de Murcia (UCAM)
- Fundacion Dr. Pedro Guillen
- G. Harold and Leila Y. Mathers Charitable Foundation
- Glenn Foundation
- Moxie Foundation
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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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