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RIKEN Baton Zone Program Cell and Gene Therapy in Ophthalmology Laboratory

Team Leader: Seiji Hori (Ph.D.)
Deputy Team Leader: Hiroshi Kiyonari (Ph.D.)

Research Summary

Team Leader: Seiji Hori Deputy Team Leader: Hiroshi Kiyonari

Vision Care Inc. has been developing regenerative medicine and gene therapy using pluripotent stem cells for patients who have lost visual function due to intractable retinal diseases.

In addition, the RIKEN Center for Biosystems Dynamics Research (BDR) has developed a large number of genetically engineered animals and new experimental animal models to analyze the pathophysiology of diseases and the efficacy of treatments.

Based on these valuable results, we aim to further optimize the technology and provide comprehensive treatment for gene therapy and regenerative medicine by advancing non-clinical animal studies specifically for retinal degenerative diseases.

[Integrated Collaborative Research with: Vision Care Inc.]

Main Research Fields

  • Medicine, Dentistry & Pharmacy

Related Research Fields

  • Biology
  • Ophthalmology
  • Gene therapy
  • Regenerative medicine

Keywords

  • Regenerative medicine
  • Pluripotent stem cell
  • Gene therapy
  • Nonclinical study
  • Retinal degeneration

Selected Publications

  • 1. Iwama Y, Nomaru H, Masuda T, Kawamura Y, Matsumura M, Murata Y, Teranishi K, Nishida K, Ota S, Mandai M, Takahashi M.
    "Label-free enrichment of human pluripotent stem cell-derived early retinal progenitor cells for cell-based regenerative therapies."
    Stem Cell Reports. 2024 Feb 13;19(2):254-269.
  • 2. Hirami Y, Mandai M, Sugita S, Maeda A, Maeda T, Yamamoto M, Uyama H, Yokota S, Fujihara M, Igeta M, Daimon T, Fujita K, Ito T, Shibatani N, Morinaga C, Hayama T, Nakamura A, Ueyama K, Ono K, Ohara H, Fujiwara M, Yamasaki Y, Watari K, Bando K, Kawabe K, Ikeda A, Kimura T, Kuwahara K, Takahashi M, Kurimoto Y.
    "Safety and stable survival of stem-cell-derived retinal organoid for 2 years in patients with retinitis pigmentosa.”
    Cell Stem Cell. 2023 Dec 7;30(12) :1585-1596
  • 3. Terada M, Kogawa Y, Shibata Y, Kitagawa M, Kato S, Iida T, Yorimitsu T, Kato A, Matsukuma K, Maeda T, Takahashi M, Kanda GN.
    "Robotic cell processing facility for clinical research of retinal cell therapy."
    SLAS Technology 2023 Dec; 28(6): 449-459
  • 4. Akishi Onishi, Yuji Tsunekawa, Michiko Mandai, Aiko Ishimaru, Yoko Ohigashi, Junki Sho, Kazushi Yasuda, Keiichiro Suzuki, Juan Carlos Izpisua Belmonte, Fumio Matsuzaki, Masayo Takahashi
    "Efficient workflow for validating homology-independent targeted integration-mediated gene insertion in rod photoreceptor cells to treat dominant-negative mutations causing retinitis pigmentosa"
    bioRxiv 2023.11.08.566127
  • 5. Mandai M, Watanabe A, Kurimoto Y, Hirami Y, Morinaga C, Daimon T, Fujihara M, Akimaru H, Sakai N, Shibata Y, Terada M, Nomiya Y, Tanishima S, Nakamura M, Kamao H, Sugita S, Onishi A, Ito T, Fujita K, Kawamata S, Go MJ, Shinohara C, Hata KI, Sawada M, Yamamoto M, Ohta S, Ohara Y, Yoshida K, Kuwahara J, Kitano Y, Amano N, Umekage M, Kitaoka F, Tanaka A, Okada C, Takasu N, Ogawa S, Yamanaka S, Takahashi M.
    "Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration."
    N Engl J Med. 2017 Mar 16;376(11):1038-1046.
  • 6. Kiyonari H, Kaneko M, Abe T, Shiraishi A, Yoshimi R, Inoue KI, Furuta Y.
    "Targeted gene disruption in a marsupial, Monodelphis domestica, by CRISPR/Cas9 genome editing."
    Curr Biol. 2021 Sep 13;31(17):3956-3963.e4.
  • 7. Nishiyama C, Saito Y, Sakaguchi A, Kaneko M, Kiyonari H, Xu Y, Arima Y, Uosaki H, Kimura W.
    "Prolonged Myocardial Regenerative Capacity in Neonatal Opossum."
    Circulation. 2022 Jul 12;146(2): 125-139.

Related Links

Lab Members

Principal investigator

Seiji Hori
Team Leader

Core members

Hiroshi Kiyonari
Deputy Team Leader
Takaya Abe
Research Scientist
Riko Yoshimi
Technical Staff II
Aki Shiraishi
Technical Staff II
Masayo Takahashi
Visiting Scientist
Hashimita Sanyal
Visiting Scientist
Tomohiro Masuda
Visiting Scientist
Kohei Homma
Visiting Scientist
Akishi Onishi
Visiting Scientist
Kazushi Yasuda
Visiting Scientist
Takesi Matsuyama Hoyos
Visiting Scientist
Tadao Maeda
Visiting Scientist
Yuki Nakano
Visiting Scientist
Mikiya Watanabe
Visiting Technician
Yoko Ohigashi
Visiting Technician
Tomoko Katayama
Visiting Technician
Takayuki Yamada
Visiting Technician
Naoko Hayashi
Visiting Technician
Junki Sho
Visiting Technician
Mitsuhiro Nishida
Visiting Technician
Hironobu Shuto
Visiting Technician
Satoshi Shirae
Visiting Technician
Masakazu Hiraoka
Visiting Technician
Atsuta Ozaki
Visiting Technician
Daiki Sakai
Visiting Technician
Kyoko Iseki
Visiting Technician
Chihiro Hayakawa
Visiting Technician
Motoki Terada
Visiting Scientist
Tomomitsu Iida
Visiting Scientist
Aoi Ishikawa
Visiting Scientist
Shinya Kato
Visiting Technician
Naomi Fujie
Visiting Technician
Miyu Koshino
Visiting Technician
Satomi Tanaka
Administrative Part-time Worker II
Naoko Ueda
Assistant

Contact Information

RIKEN Center for Biosystems Dynamics Research
2-2-3 Minatojima Minami-machi, Chuo-ku,
Kobe, Hyogo 650-0047, Japan
Email: ks-rinc [at] ml.riken.jp

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