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

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 re...

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Veröffentlicht in:Stem cell research 2021-05, Vol.53, p.102360-102360, Article 102360
Hauptverfasser: Kato, Hisaya, Maezawa, Yoshiro, Ouchi, Yasuo, Takayama, Naoya, Sone, Masamitsu, Sone, Kanako, Takada-Watanabe, Aki, Tsujimura, Kyoko, Koshizaka, Masaya, Nagasawa, Sayaka, Saitoh, Hisako, Ohtaka, Manami, Nakanishi, Mahito, Tahara, Hidetoshi, Shimamoto, Akira, Iwama, Atsushi, Eto, Koji, Yokote, Koutaro
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Sprache:eng
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Zusammenfassung: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.
ISSN:1873-5061
1876-7753
DOI:10.1016/j.scr.2021.102360