Single-dose CRISPR-Cas9 therapy extends lifespan of mice with Hutchinson-Gilford progeria syndrome.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 30778240.
- Also identified by DOI 10.1038/s41591-019-0343-4 and PMC identifier 6541418.
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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.
Medical subject headings
- CRISPR-Cas Systems
- Genetic Therapy
- Lamin Type A
- Longevity
- Progeria