Ben M'Barek Karim, Monville Christelle
INSERM U861, I-Stem, AFM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, 91100 Corbeil-Essonnes, France.
UEVE U861, I-Stem, AFM, Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, 91100 Corbeil-Essonnes, France.
Stem Cells Int. 2019 Jan 23;2019:4568979. doi: 10.1155/2019/4568979. eCollection 2019.
Retinal degeneration is an irreversible phenomenon caused by various disease conditions including age-related macular degeneration (AMD) and retinitis pigmentosa (RP). During the course of these diseases, photoreceptors (PRs) are susceptible to degeneration due to their malfunctions or to a primary dysfunction of the retinal pigment epithelium (RPE). Once lost, these cells could not be endogenously regenerated in humans, and cell therapy to replace the lost cells is one of the promising strategies to recover vision. Depending on the nature of the primary defect and the stage of the disease, RPE cells, PRs, or both might be transplanted to achieve therapeutic effects. We describe in this review the current knowledge and recent progress to develop such approaches. The different cell sources proposed for cell therapy including human pluripotent stem cells are presented with their advantages and limits. Another critical aspect described herein is the pharmaceutical formulation of the end product to be delivered into the eye of patients. Finally, we also outline the future research directions in order to develop a complex multilayered retinal tissue for end-stage patients.
视网膜变性是一种由多种疾病状况引起的不可逆现象,包括年龄相关性黄斑变性(AMD)和视网膜色素变性(RP)。在这些疾病的发展过程中,光感受器(PRs)由于其功能异常或视网膜色素上皮(RPE)的原发性功能障碍而容易发生变性。一旦这些细胞丢失,人类体内无法内源性再生,而细胞疗法来替代丢失的细胞是恢复视力的一种有前景的策略。根据原发性缺陷的性质和疾病阶段,可能会移植RPE细胞、PRs或两者以达到治疗效果。我们在本综述中描述了开发此类方法的当前知识和最新进展。提出用于细胞治疗的不同细胞来源,包括人类多能干细胞,并介绍了它们的优点和局限性。本文描述的另一个关键方面是待递送至患者眼中的最终产品的药物制剂。最后,我们还概述了未来的研究方向,以便为晚期患者开发复杂的多层视网膜组织。