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。

Personalized Medicine: Cell and Gene Therapy Based on Patient-Specific iPSC-Derived Retinal Pigment Epithelium Cells.

作者信息

Li Yao, Chan Lawrence, Nguyen Huy V, Tsang Stephen H

机构信息

Department of Ophthalmology, Columbia University Medical Center, Columbia University, 160 Fort Washington Ave, Research Annex, Room 513, 10032, New York, NY, USA.

Columbia University College of Physicians and Surgeons, 100 Haven Ave, Apt 14B, 10032, New York, NY, USA.

出版信息

Adv Exp Med Biol. 2016;854:549-55. doi: 10.1007/978-3-319-17121-0_73.

DOI:10.1007/978-3-319-17121-0_73

PMID:26427458

Abstract

Interest in generating human induced pluripotent stem (iPS) cells for stem cell modeling of diseases has overtaken that of patient-specific human embryonic stem cells due to the ethical, technical, and political concerns associated with the latter. In ophthalmology, researchers are currently using iPS cells to explore various applications, including: (1) modeling of retinal diseases using patient-specific iPS cells; (2) autologous transplantation of differentiated retinal cells that undergo gene correction at the iPS cell stage via gene editing tools (e.g., CRISPR/Cas9, TALENs and ZFNs); and (3) autologous transplantation of patient-specific iPS-derived retinal cells treated with gene therapy. In this review, we will discuss the uses of patient-specific iPS cells for differentiating into retinal pigment epithelium (RPE) cells, uncovering disease pathophysiology, and developing new treatments such as gene therapy and cell replacement therapy via autologous transplantation.

摘要

由于与患者特异性人类胚胎干细胞相关的伦理、技术和政治问题，利用人类诱导多能干细胞（iPS细胞）进行疾病干细胞建模的研究兴趣已超过了对患者特异性人类胚胎干细胞的研究兴趣。在眼科领域，研究人员目前正在使用iPS细胞探索各种应用，包括：（1）使用患者特异性iPS细胞对视网膜疾病进行建模；（2）通过基因编辑工具（如CRISPR/Cas9、转录激活因子样效应物核酸酶和锌指核酸酶）在iPS细胞阶段进行基因校正后对分化的视网膜细胞进行自体移植；以及（3）对经基因治疗处理的患者特异性iPS衍生视网膜细胞进行自体移植。在本综述中，我们将讨论患者特异性iPS细胞在分化为视网膜色素上皮（RPE）细胞、揭示疾病病理生理学以及开发新治疗方法（如通过自体移植进行基因治疗和细胞替代治疗）方面的用途。