PDF(Pubmed)
Abstract：
Photoreceptor degeneration is a major cause of untreatable blindness worldwide and has recently been targeted by emerging technologies, including cell- and gene-based therapies. Cell types of neural lineage have shown promise for replacing either photoreceptors or retinal pigment epithelial cells following delivery to the subretinal space, while cells of bone marrow lineage have been tested for retinal trophic effects following delivery to the vitreous cavity. Here we explore an alternate approach in which cells from the immature neural retinal are delivered to the vitreous cavity with the goal of providing trophic support for degenerating photoreceptors. Rat and human retinal progenitor cells were transplanted to the vitreous of rats with a well-studied photoreceptor dystrophy, resulting in substantial anatomical preservation and functional rescue of vision. This work provides scientific proof-of-principle for a novel therapeutic approach to photoreceptor degeneration that is currently being evaluated in clinical trials.
摘要：
光感受器变性是全球范围内无法治愈的失明的主要原因，最近已成为新兴技术的目标，包括基于细胞和基因的疗法。神经谱系的细胞类型已显示出有望在递送到视网膜下隙后替代光感受器或视网膜色素上皮细胞。而骨髓细胞在递送到玻璃体腔后已经测试了视网膜营养作用。在这里，我们探索了一种替代方法，其中未成熟神经视网膜的细胞被输送到玻璃体腔，目的是为变性的光感受器提供营养支持。将大鼠和人视网膜祖细胞移植到患有充分研究的光感受器营养不良的大鼠的玻璃体中，导致大量的解剖保存和视力的功能挽救。这项工作为目前正在临床试验中评估的光感受器变性的新型治疗方法提供了科学的原理证明。
