Pluripotent Stem Cell-Based Organoid Technologies for Developing Next-Generation Vision Restoration Therapies of Blindness.
Pluripotent Stem Cell-Based Organoid Technologies for Developing Next-Generation Vision Restoration Therapies of Blindness.
复制标题
DOI：
10.1089/jop.2020.0016
复制
发表时间：
2020-10
期刊：
影响因子：
--
通讯作者：
Ratnesh K Singh;F. Binette;M. Seiler;S. Petersen-Jones;I. Nasonkin
中科院分区：
文献类型：
--
作者：
Ratnesh K Singh;F. Binette;M. Seiler;S. Petersen-Jones;I. Nasonkin
与眼睛后部视网膜细胞死亡相关的失明是由许多高患病率的病症引起的，如青光眼、年龄相关性黄斑变性和糖尿病视网膜病变。此外，大量的孤儿遗传（大多数是单基因的）条件，如视网膜色素变性和利伯先天性黑蒙，增加了致盲视网膜变性疾病患者的总数。由视网膜退化和丧失引起的失明迄今为止是无法治愈的。利用分子和再生医学方法的现代生物医学研究有许多突破性的发现和动物失明的原理性治疗方法。然而，这些方法作为治疗方法标准化和商业化的速度很慢，以使由于视网膜变性病症而失去视力的人受益。在这篇综述中，我们将概述主要的再生医学方法，这是新兴的有希望的保存或/和恢复视力。我们讨论了这些方法中的每一种达到商业化步骤并转化为治疗的潜力，这至少可以改善由视网膜细胞死亡引起的失明。
Blindness, associated with death of retinal cells at the back of the eye, is caused by a number of conditions with high prevalence such as glaucoma, age-related macular degeneration, and diabetic retinopathy. In addition, a large number of orphan inherited (mostly monogenic) conditions, such as retinitis pigmentosa and Leber Congenital Amaurosis, add to the overall number of patients with blinding retinal degenerative diseases. Blindness caused by deterioration and loss of retina is so far incurable. Modern biomedical research leveraging molecular and regenerative medicine approaches had a number of groundbreaking discoveries and proof-of-principle treatments of blindness in animals. However, these methods are slow to be standardized and commercialized as therapies to benefit people losing their eyesight due to retinal degenerative conditions. In this review, we will outline major regenerative medicine approaches, which are emerging as promising for preserving or/and restoring vision. We discuss the potential of each of these approaches to reach commercialization step and be converted to treatments, which could at least ameliorate blindness caused by retinal cell death.