干细胞移植治疗视网膜变性疾病的基础和临床研究进展.docVIP

干细胞移植治疗视网膜变性疾病面临的挑战和前景 Translation of stem cell-based therapies for retinal degeneration Yin Zhengqin, Liu Yong (Southwest Hospital, Southwest Eye Hospital, Third Military Medical University, Chongqing, 400038, China) [Abstract] Present therapies for retinal degenerative diseases are limited in their efficacy. Consequently, the development of novel neuroprotective and neuroregenerative treatments is a top priority. Stem cells hold great promise for regenerative medicine, and recently many efforts have been devoted to find suitable candidates for retinal transplants. A variety of stem-cell-based approaches are being advanced to replace impaired retinal cells and/or to slow the disease processes by providing neuroprotective factors, with the ultimate aim of long-term improvement in visual function. Furthermore, clinical trials are in the early stages, and data on safety and efficacy are widely anticipated. We discuss here the challenges and potential of stem-cell-based therapies in treatment of retinal blindness. [Key words] retinal degeneration; stem cell therapy; translational medicine Supported by the National Basic Research Program (973 Program, 2013CB967000). Corresponding author, Yin Zhengqin, Tel: 86-23 E-mail: qinzyin@ [基金项目] 国家重点基础研究发展计划（973计划，2013CB967000） [通信作者] 阴正勤，女，博士，主任医师，教授，博士生导师。担任中华医学会眼科学会副主任委员，中华医学会眼科学分会视觉生理学组组长，全军眼科学会主任委员，中国视力残疾康复专业委员会副主任委员，国家自然科学基金重点项目评审委员会委员。获国家重点基础研究发展计划（973计划）课题（首席科学家）、国家自然科学基金重点课题等20余项。获军队科技进步二等奖（1999）、重庆市科技进步一等奖（2007），亚太眼科学会防盲杰出贡献奖qinzyin@ [优先出版] /kcms/detail/51.1095.R1107.002.html（2013-06-27） 视网膜变性类疾病主要包括视网膜色素变性(retinitis pigmentosa，RP)age related macular degeneration, AMD），在东亚国家，还有一种发病率较高的特殊类型，即高度近视引起的视网膜变性。这类疾病的共同特点在于视网膜中的关键细胞（如感光细胞变性、死亡，由于变性和凋亡，移植健康的视网膜组织或者细胞，成为目前最具前景的治疗策略[1]。Royol等[2]开展了第1例同种异体胚胎视网膜移植试验，近20年人们相继进行了视网膜色素上皮细胞、视网膜感光细胞、胚胎干细胞、骨髓间充质干细胞、视网膜干细胞、胚胎视网膜神经上皮层、胚胎全层视网膜(色素上皮+神经上皮)以及视网膜芯片、视网膜假体移植到动物的视网膜下间隙的研究[3-6]üller细胞的逆分化[7-8]。理论上，建立有效的新的突触联系和在此基础上的视功能改善，是移植成功的标准[9]Maclaren等[10]及2008年Bartsc