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Generation of disease-specific and CRISPR/Cas9-mediated gene-corrected iPS cells from a patient with adult progeria Werner syndrome.
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.
Affiliation
  • Kato H; Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, Chiba, Japan; Division of Diabetes, Metabolism and Endocrinology, Chiba University Hospital, Chiba, Japan. Electronic address: hisayakato@chiba-u.jp.
  • Maezawa Y; Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, Chiba, Japan; Division of Diabetes, Metabolism and Endocrinology, Chiba University Hospital, Chiba, Japan. Electronic address: yoshiromaezawa@chiba-u.jp.
  • Ouchi Y; Department of Regenerative Medicine, Chiba University Graduate School of Medicine, Chiba, Japan; Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA, USA.
  • Takayama N; Department of Regenerative Medicine, Chiba University Graduate School of Medicine, Chiba, Japan.
  • Sone M; Hibernation Metabolism, Physiology and Development Group, Institute of Low Temperature Science, Hokkaido University, Sapporo, Japan.
  • Sone K; Hibernation Metabolism, Physiology and Development Group, Institute of Low Temperature Science, Hokkaido University, Sapporo, Japan.
  • Takada-Watanabe A; Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, Chiba, Japan.
  • Tsujimura K; Department of Regenerative Medicine, Chiba University Graduate School of Medicine, Chiba, Japan.
  • Koshizaka M; Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, Chiba, Japan; Division of Diabetes, Metabolism and Endocrinology, Chiba University Hospital, Chiba, Japan.
  • Nagasawa S; Department of Legal Medicine, Graduate School of Medicine, Chiba University, Chiba, Japan.
  • Saitoh H; Department of Legal Medicine, Graduate School of Medicine, Chiba University, Chiba, Japan.
  • Ohtaka M; TOKIWA-Bio, Inc., Tsukuba, Japan; National Institute of Advanced Industrial Science and Technology (AIST), Tsukuba, Japan.
  • Nakanishi M; TOKIWA-Bio, Inc., Tsukuba, Japan; National Institute of Advanced Industrial Science and Technology (AIST), Tsukuba, Japan.
  • Tahara H; Department of Cellular and Molecular Biology, Graduate School of Biomedical and Health Sciences, Hiroshima University, Hiroshima, Japan.
  • Shimamoto A; Department of Regenerative Medicine Research, Faculty of Pharmaceutical Sciences, Sanyo-Onoda City University, Sanyo-Onoda, Yamaguchi, Japan.
  • Iwama A; Division of Stem Cell and Molecular Medicine, Center for Stem Cell Biology and Regenerative Medicine, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan.
  • Eto K; Department of Regenerative Medicine, Chiba University Graduate School of Medicine, Chiba, Japan; Department of Clinical Application, Center for iPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan.
  • Yokote K; Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, Chiba, Japan; Division of Diabetes, Metabolism and Endocrinology, Chiba University Hospital, Chiba, Japan. Electronic address: kyokote@faculty.chiba-u.jp.
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.
Subject(s)

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

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