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AAV-mediated editing of PMP22 rescues Charcot-Marie-Tooth disease type 1A features in patient-derived iPS Schwann cells.
Yoshioka, Yuki; Taniguchi, Juliana Bosso; Homma, Hidenori; Tamura, Takuya; Fujita, Kyota; Inotsume, Maiko; Tagawa, Kazuhiko; Misawa, Kazuharu; Matsumoto, Naomichi; Nakagawa, Masanori; Inoue, Haruhisa; Tanaka, Hikari; Okazawa, Hitoshi.
Affiliation
  • Yoshioka Y; Department of Neuropathology, Medical Research Institute, Tokyo Medical and Dental University, 1-5-45, Yushima, Bunkyo-ku, Tokyo, 113-8510, Japan.
  • Taniguchi JB; Department of Neuropathology, Medical Research Institute, Tokyo Medical and Dental University, 1-5-45, Yushima, Bunkyo-ku, Tokyo, 113-8510, Japan.
  • Homma H; Department of Neuropathology, Medical Research Institute, Tokyo Medical and Dental University, 1-5-45, Yushima, Bunkyo-ku, Tokyo, 113-8510, Japan.
  • Tamura T; Department of Neuropathology, Medical Research Institute, Tokyo Medical and Dental University, 1-5-45, Yushima, Bunkyo-ku, Tokyo, 113-8510, Japan.
  • Fujita K; Department of Neuropathology, Medical Research Institute, Tokyo Medical and Dental University, 1-5-45, Yushima, Bunkyo-ku, Tokyo, 113-8510, Japan.
  • Inotsume M; Department of Neuropathology, Medical Research Institute, Tokyo Medical and Dental University, 1-5-45, Yushima, Bunkyo-ku, Tokyo, 113-8510, Japan.
  • Tagawa K; Department of Neuropathology, Medical Research Institute, Tokyo Medical and Dental University, 1-5-45, Yushima, Bunkyo-ku, Tokyo, 113-8510, Japan.
  • Misawa K; Department of Human Genetics, Yokohama City University Graduate School of Medicine, Yokohama, Kanagawa, 236-0004, Japan.
  • Matsumoto N; RIKEN Center for Advanced Intelligence Project, 1-4-1 Nihonbashi, Chuo-ku, Tokyo, 103-0027, Japan.
  • Nakagawa M; Department of Human Genetics, Yokohama City University Graduate School of Medicine, Yokohama, Kanagawa, 236-0004, Japan.
  • Inoue H; Department of Neurology, Kyoto Prefectural University of Medicine, Kyoto, 606-8507, Japan.
  • Tanaka H; Center for iPS Cell Research and Application (CiRA), Kyoto University, Kyoto, 606-8507, Japan.
  • Okazawa H; Drug-discovery cellular basis development team, RIKEN BioResource Center, Kyoto, 606-8507, Japan.
Commun Med (Lond) ; 3(1): 170, 2023 Nov 28.
Article in En | MEDLINE | ID: mdl-38017287
Charcot-Marie-Tooth disease type 1A (CMT1A) is a common heritable form of the condition that develops when nerves in the body's extremities, such as the hands, feet and arms, are damaged due to an extra copy of PMP22 gene being incorrectly produced. Currently, no known therapies exist. Here, we developed a method to delete the additional copy of PMP22 gene by 20­40% to prevent overproduction. Our results show that this method can reduce PMP22 protein production, leading to near normal production in patient's nerve cells. Further safety assessments should now be undertaken. If the treatment is safe for patients it could become a therapeutic option for CMT1A patients.
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Commun Med (Lond) Year: 2023 Document type: Article Affiliation country: Japan Country of publication: United kingdom
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Commun Med (Lond) Year: 2023 Document type: Article Affiliation country: Japan Country of publication: United kingdom