Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome.
Olaya Santiago-FernándezFernando G OsorioVíctor QuesadaFrancisco RodríguezSammy BassoDaniel MaesoLoïc RolasAnna BarkawaySussan NoursharghAlicia R FolguerasJosé M P FreijeCarlos López-OtínPublished in: Nature medicine (2019)
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.
Keyphrases
- crispr cas
- genome editing
- genome wide
- copy number
- case report
- induced apoptosis
- dna methylation
- gene expression
- type diabetes
- muscular dystrophy
- cell cycle arrest
- signaling pathway
- adipose tissue
- cell proliferation
- big data
- high fat diet induced
- climate change
- insulin resistance
- skeletal muscle
- genome wide identification
- deep learning