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Generation of disease-specific and CRISPR/Cas9-mediated gene-corrected iPS cells from a patient with adult progeria Werner syndrome

Authors :
Koutaro Yokote
Koji Eto
Hisaya Kato
Atsushi Iwama
Mahito Nakanishi
Masamitsu Sone
Kyoko Tsujimura
Akira Shimamoto
Naoya Takayama
Hisako Saitoh
Masaya Koshizaka
Aki Takada-Watanabe
Sayaka Nagasawa
Manami Ohtaka
Yasuo Ouchi
Kanako Sone
Hidetoshi Tahara
Yoshiro Maezawa
Source :
Stem Cell Research, Vol 53, Iss, Pp 102360-(2021)
Publication Year :
2021

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.

Details

ISSN :
18767753
Volume :
53
Database :
OpenAIRE
Journal :
Stem cell research
Accession number :
edsair.doi.dedup.....d54d77cfa264beb3221f0ee2f25e7e50