Osakada Fumitaka
Laboratory of Cellular Pharmacology, Graduate School of Pharmaceutical Sciences, Nagoya University.
Yakugaku Zasshi. 2017;137(1):23-29. doi: 10.1248/yakushi.16-00225.
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.
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。