Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 30778239.
- Also identified by DOI 10.1038/s41591-018-0338-6 and PMC identifier 6546610.
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Abstract
CRISPR/Cas9-based therapies hold considerable promise for the treatment of genetic diseases. Among these, Hutchinson-Gilford progeria syndrome, caused by a point mutation in the LMNA gene, stands out as a potential candidate. Here, we explore the efficacy of a CRISPR/Cas9-based approach that reverts several alterations in Hutchinson-Gilford progeria syndrome cells and mice by introducing frameshift mutations in the LMNA gene.
Medical subject headings
- CRISPR-Cas Systems
- Genetic Therapy
- Lamin Type A
- Progeria