Generation of disease-specific and CRISPR/Cas9-mediated gene-corrected iPS cells from a patient with adult progeria Werner syndrome
Generation of disease-specific and CRISPR/Cas9-mediated gene-corrected iPS cells from a patient with adult progeria Werner syndrome
Blog Article
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 Tobacco family and is predominantly involved in DNA replication, repair, and Coverless Duvet 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.