Stem Cell Research (May 2021)
Generation of disease-specific and CRISPR/Cas9-mediated gene-corrected iPS cells from a patient with adult progeria Werner syndrome
- 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
Affiliations
- Hisaya Kato
- 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; Corresponding authors at: Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, 1-8-1 Inohana, Chuo-ku, Chiba, 260-8670, Japan.
- Yoshiro Maezawa
- 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; Corresponding authors at: Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, 1-8-1 Inohana, Chuo-ku, Chiba, 260-8670, Japan.
- Yasuo Ouchi
- Department of Regenerative Medicine, Chiba University Graduate School of Medicine, Chiba, Japan; Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA, USA
- Naoya Takayama
- Department of Regenerative Medicine, Chiba University Graduate School of Medicine, Chiba, Japan
- Masamitsu Sone
- Hibernation Metabolism, Physiology and Development Group, Institute of Low Temperature Science, Hokkaido University, Sapporo, Japan
- Kanako Sone
- Hibernation Metabolism, Physiology and Development Group, Institute of Low Temperature Science, Hokkaido University, Sapporo, Japan
- Aki Takada-Watanabe
- Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, Chiba, Japan
- Kyoko Tsujimura
- Department of Regenerative Medicine, Chiba University Graduate School of Medicine, Chiba, Japan
- Masaya Koshizaka
- 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
- Sayaka Nagasawa
- Department of Legal Medicine, Graduate School of Medicine, Chiba University, Chiba, Japan
- Hisako Saitoh
- Department of Legal Medicine, Graduate School of Medicine, Chiba University, Chiba, Japan
- Manami Ohtaka
- TOKIWA-Bio, Inc., Tsukuba, Japan; National Institute of Advanced Industrial Science and Technology (AIST), Tsukuba, Japan
- Mahito Nakanishi
- TOKIWA-Bio, Inc., Tsukuba, Japan; National Institute of Advanced Industrial Science and Technology (AIST), Tsukuba, Japan
- Hidetoshi Tahara
- Department of Cellular and Molecular Biology, Graduate School of Biomedical and Health Sciences, Hiroshima University, Hiroshima, Japan
- Akira Shimamoto
- Department of Regenerative Medicine Research, Faculty of Pharmaceutical Sciences, Sanyo-Onoda City University, Sanyo-Onoda, Yamaguchi, Japan
- Atsushi Iwama
- 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
- Koji Eto
- 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
- Koutaro Yokote
- 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; Corresponding authors at: Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, 1-8-1 Inohana, Chuo-ku, Chiba, 260-8670, Japan.
- Journal volume & issue
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Vol. 53
p. 102360
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.
