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

从患有成年早衰症沃纳综合征的患者体内生成疾病特异性和 CRISPR/Cas9 介导的基因校正的 iPS 细胞

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作者:Hisaya Kato, Yoshiro Maezawa, Yasuo Ouchi, Naoya Takayama, Masamitsu Sone, Kanako Sone, Aki Takada-Watanabe, Kyoko Tsujimura, Masaya Koshizaka, Sayaka Nagasawa, Hisako Saitoh, Manami Ohtaka, Mahito Nakanishi, Hidetoshi Tahara, Akira Shimamoto, Atsushi Iwama, Koji Eto, Koutaro Yokote

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.

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