Safety and stable survival of stem-cell-derived retinal organoid for 2 years in patients with retinitis pigmentosa

被引：22

作者：

Hirami, Yasuhiko ^{[1
,2
,3
]}

Mandai, Michiko ^{[1
,3
,4
,5
]}

Sugita, Sunao ^{[1
,3
]}

Maeda, Akiko ^{[1
,3
,4
]}

Maeda, Tadao ^{[1
,4
]}

Yamamoto, Midori ^{[1
,4
]}

Uyama, Hirofumi ^{[1
,2
]}

Yokota, Satoshi ^{[1
,2
,3
]}

Fujihara, Masashi ^{[1
,2
,3
]}

Igeta, Masataka ^{[6
]}

Daimon, Takashi ^{[6
]}

Fujita, Kanako ^{[4
]}

Ito, Tomoko ^{[4
]}

Shibatani, Naoki ^{[4
]}

Morinaga, Chikako ^{[5
]}

Hayama, Tetsuya

Nakamura, Aya ^{[8
]}

Ueyama, Kazuki ^{[8
]}

Ono, Keiichi ^{[8
]}

Ohara, Hidetaka ^{[9
]}

Fujiwara, Masayo ^{[7
]}

Yamasaki, Suguru ^{[7
]}

Watari, Kenji ^{[7
]}

Bando, Kiyoko ^{[9
]}

Kawabe, Keigo ^{[9
]}

Ikeda, Atsushi ^{[7
]}

Kimura, Toru ^{[7
,9
]}

Kuwahara, Atsushi ^{[7
]}

Takahashi, Masayo ^{[1
]}

Kurimoto, Yasuo ^{[1
,2
,3
]}

机构：

[1] Kobe City Eye Hosp, Dept Ophthalmol, Kobe 6500047, Japan

[2] Kobe City Med Ctr Gen Hosp, Dept Ophthalmol, Kobe 6500047, Japan

[3] RIKEN Ctr Biosyst Dynam Res, Lab Retinal Regenerat, Kobe 6500047, Japan

[4] Kobe City Eye Hosp, Res Ctr, Kobe 6500047, Japan

[5] RIKEN Program Drug Discovery & Med Technol Platfor, Yokohama 2300045, Japan

[6] Hyogo Med Univ, Dept Biostat, Nishinomiya 6638501, Japan

[7] Sumitomo Pharm Co Ltd, Regenerat & Cellular Med Kobe Ctr, Kobe 6500047, Japan

[8] Sumitomo Pharm Co Ltd, Technol Res & Dev Div, Kobe 6500047, Japan

[9] Sumitomo Pharm Co Ltd, Regenerat & Cellular Med Off, Tokyo 1036012, Japan

来源：

CELL STEM CELL | 2023年 / 30卷 / 12期

关键词：

TRANSPLANTATION; VISION; SHEETS;

D O I：

10.1016/j.stem.2023.11.004

中图分类号：

Q813 [细胞工程];

学科分类号：

摘要：

Transplantation of induced pluripotent stem cell (iPSC)-derived retinal organoids into retinal disease animal models has yielded promising results, and several clinical trials on iPSC-derived retinal pigment epithelial cell transplantation have confirmed its safety. In this study, we performed allogeneic iPSC-derived retinal orga-noid sheet transplantation in two subjects with advanced retinitis pigmentosa (jRCTa050200027). The pri-mary endpoint was the survival and safety of the transplanted retinal organoid sheets in the first year post-transplantation. The secondary endpoints were the safety of the transplantation procedure and visual function evaluation. The grafts survived in a stable condition for 2 years, and the retinal thickness increased at the transplant site without serious adverse events in both subjects. Changes in visual function were less pro-gressive than those of the untreated eye during the follow-up. Allogeneic iPSC-derived retinal organoid sheet transplantation is a potential therapeutic approach, and the treatment's safety and efficacy for visual function should be investigated further.

引用

页码：1585 / 1596.e6

页数：19

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