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[用于视网膜再生医学的细胞和组织基产品的开发]

[Development of Cellular and Tissue-based Products for Retinal Regenerative Medicine].

作者信息

Osakada Fumitaka

机构信息

Laboratory of Cellular Pharmacology, Graduate School of Pharmaceutical Sciences, Nagoya University.

出版信息

Yakugaku Zasshi. 2017;137(1):23-29. doi: 10.1248/yakushi.16-00225.

DOI:10.1248/yakushi.16-00225
PMID:28049891
Abstract

Since the discovery of induced pluripotent stem cells (iPSCs) generation, much progress has been made in the fields of medical and pharmaceutical research, such as cell transplantation therapy. We have generated retinal cells and tissues, including retinal pigment epithelia (RPE), from human iPSCs. The ability to produce iPSCs from patients allows for autologous transplantation without causing immune rejection. The autologous transplantation of iPSC-derived retinal pigment epithelial sheets to a patient with age-related macular degeneration was carried out in Japan in 2014 as a first-in-human clinical study. Biotechnology has enabled the development of a wide range of drugs, including cell-based drugs. We are currently developing iPSC-derived RPE sheets as next-generation cell-based drugs aimed at allogeneic transplantation utilizing iPSC banks of homozygotes of human leukocyte antigen at 3 loci. Regulatory science concerning cellular and tissue-based products is a vital matter associated with the realization of regenerative medicine. Here we review the most recent progress in retinal regeneration and drug development, as well as its future prospects.

摘要

自从诱导多能干细胞(iPSC)产生技术被发现以来,医学和药物研究领域取得了很大进展,比如细胞移植疗法。我们已经从人iPSC中生成了视网膜细胞和组织,包括视网膜色素上皮(RPE)。从患者身上产生iPSC的能力使得自体移植成为可能,且不会引起免疫排斥。2014年在日本进行了首例将iPSC来源的视网膜色素上皮片移植给一名年龄相关性黄斑变性患者的人体临床研究。生物技术推动了包括基于细胞的药物在内的多种药物的研发。我们目前正在将iPSC来源的RPE片开发为下一代基于细胞的药物,旨在利用人类白细胞抗原3个位点纯合子的iPSC库进行同种异体移植。关于细胞和组织产品的监管科学是与再生医学的实现相关的重要事项。在此,我们综述视网膜再生和药物开发的最新进展及其未来前景。

相似文献

1
[Development of Cellular and Tissue-based Products for Retinal Regenerative Medicine].[用于视网膜再生医学的细胞和组织基产品的开发]
Yakugaku Zasshi. 2017;137(1):23-29. doi: 10.1248/yakushi.16-00225.
2
Fabricating retinal pigment epithelial cell sheets derived from human induced pluripotent stem cells in an automated closed culture system for regenerative medicine.在自动化封闭培养系统中制造源自人诱导多能干细胞的视网膜色素上皮细胞片用于再生医学。
PLoS One. 2019 Mar 13;14(3):e0212369. doi: 10.1371/journal.pone.0212369. eCollection 2019.
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Induced pluripotent stem cell-based therapy for age-related macular degeneration.基于诱导多能干细胞的年龄相关性黄斑变性治疗
Expert Opin Biol Ther. 2017 Sep;17(9):1113-1126. doi: 10.1080/14712598.2017.1346079. Epub 2017 Jun 30.
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Pluripotent Stem Cells in Clinical Cell Transplantation: Focusing on Induced Pluripotent Stem Cell-Derived RPE Cell Therapy in Age-Related Macular Degeneration.临床细胞移植中的多能干细胞:聚焦于诱导多能干细胞衍生的 RPE 细胞治疗年龄相关性黄斑变性。
Int J Mol Sci. 2022 Nov 9;23(22):13794. doi: 10.3390/ijms232213794.
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[Retinal regeneration with iPS cells ‒ Clinical trials for retinal degenerative disorders].[诱导多能干细胞用于视网膜再生——视网膜退行性疾病的临床试验]
Nihon Rinsho Meneki Gakkai Kaishi. 2015;38(2):79-85. doi: 10.2177/jsci.38.79.
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Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration.自体诱导干细胞衍生的视网膜细胞治疗黄斑变性。
N Engl J Med. 2017 Mar 16;376(11):1038-1046. doi: 10.1056/NEJMoa1608368.
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Personalized Medicine: Cell and Gene Therapy Based on Patient-Specific iPSC-Derived Retinal Pigment Epithelium Cells.个性化医疗:基于患者特异性诱导多能干细胞衍生的视网膜色素上皮细胞的细胞和基因疗法。
Adv Exp Med Biol. 2016;854:549-55. doi: 10.1007/978-3-319-17121-0_73.
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Tumorigenicity studies of induced pluripotent stem cell (iPSC)-derived retinal pigment epithelium (RPE) for the treatment of age-related macular degeneration.诱导多能干细胞(iPSC)衍生的视网膜色素上皮(RPE)治疗年龄相关性黄斑变性的致瘤性研究。
PLoS One. 2014 Jan 14;9(1):e85336. doi: 10.1371/journal.pone.0085336. eCollection 2014.
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Reproducible production and image-based quality evaluation of retinal pigment epithelium sheets from human induced pluripotent stem cells.从人诱导多能干细胞中可重现生产和基于图像的视网膜色素上皮片的质量评估。
Sci Rep. 2020 Sep 1;10(1):14387. doi: 10.1038/s41598-020-70979-y.
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iPSC-RPE in Retinal Degeneration: Recent Advancements and Future Perspectives.诱导多能干细胞(iPSC)来源的视网膜色素上皮(RPE)在视网膜变性中的应用:最新进展和未来展望。
Cold Spring Harb Perspect Med. 2023 Aug 1;13(8):a041308. doi: 10.1101/cshperspect.a041308.

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The role of preservation in the variability of regenerative medicine products.保存对再生医学产品变异性的作用。
Regen Eng Transl Med. 2019;5(4):323-331. doi: 10.1007/s40883-019-00110-9. Epub 2019 May 31.
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Paracrine effects of intraocularly implanted cells on degenerating retinas in mice.眼内移植细胞对小鼠变性视网膜的旁分泌作用。
Stem Cell Res Ther. 2020 Mar 31;11(1):142. doi: 10.1186/s13287-020-01651-5.