Generation of disease-specific and CRISPR/Cas9-mediated gene-corrected iPS cells from a patient with adult progeria Werner syndrome.
Stem Cell Res
; 53: 102360, 2021 05.
Article
in En
| MEDLINE
| ID: mdl-34087989
ABSTRACT
Adult progeria Werner syndrome (WS), a rare autosomal recessive disorder, is characterized by accelerated aging symptoms after puberty. The causative gene, WRN, is a member of the RecQ DNA helicase family and is predominantly involved in DNA replication, repair, and telomere maintenance. Here, we report the generation of iPS cells from a patient with WS and correction of the WRN gene by the CRISPR/Cas9-mediated method. These iPSC lines would be a valuable resource for deciphering the pathogenesis of WS.
Full text:
1
Database:
MEDLINE
Main subject:
Werner Syndrome
/
Induced Pluripotent Stem Cells
Limits:
Adult
/
Humans
Language:
En
Journal:
Stem Cell Res
Year:
2021
Type:
Article