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
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