重视前沿生物技术在视网膜退行性疾病中的整合应用  

Emphasis on the integration of advanced biological technology in retinal degenerative diseases

作　　者：张敬学[1] 卢清君[1] 

机构地区：[1]首都医科大学附属北京同仁医院 北京同仁眼科中心 北京市眼科学与视觉科学重点实验室,100730

出　　处：《中华眼科医学杂志（电子版）》2015年第5期1-8,共8页Chinese Journal of Ophthalmologic Medicine(Electronic Edition)

基　　金：北京市优秀人才-青年骨干项目基金(编号:2014000021469G262);北京市眼科研究所引领计划基金(编号:201519)

摘　　要：视网膜退行性疾病,如视网膜色素变性(RP)、年龄相关性黄斑变性(AMD)、以及青光眼等疾病的主要病理基础是视网膜各级神经元的结构和功能性异常,最终造成患者视力的不可逆性损害,是严重的致盲性眼病,目前缺乏有效的治疗方法。随着近年来科学技术的飞速发展,基因治疗、干细胞治疗、自适应光学技术以及人工视觉给上述疾病的治疗提供了可能。人类基因组计划的完成及相关测序技术的广泛应用,为人类对疾病的本质认识带来了新的革命,也为疾病的分子分型、生物诊断及基因水平的治疗都带来了新的动力。人们对眼病的了解也越来越全面,治疗方案在基因替代治疗基础上,进一步整合了细胞因子治疗以及光遗传学治疗等。基因治疗技术在眼科取得了巨大的进展,但不能阻止某些有功能的细胞进一步凋亡,这就需要我们联合一类可以挽救细胞凋亡的基因共同移植,要么需要采用细胞替代治疗,将有功能的细胞代替损伤的细胞,从而恢复其功能。由于干细胞无限的增生能力和多向的分化潜能,成为了细胞替代治疗的新希望。通过视网膜干细胞移植,使其整合入视网膜各层并且分化为目标细胞,以重建视网膜功能,给不可逆性盲眼患者带来了曙光。神经干细胞的自我更新、多分化潜能,使得其非常适合与细胞移植;而机体内广泛存在的神经干细胞加上体外培养扩增技术为我们提供了充足的细胞源。但干细胞的眼部移植后细胞存在移行能力、存活效率以及向目的细胞的分化能力等难点,而基因治疗技术和纳米生物学材料的发展为解决这一难题提供了新的思路,更进一步的推动了这一技术的发展。近年来,自适应光学技术在眼科领域的应用逐步扩大,有望实现各种眼病的早期诊断和治疗,把疾病消灭在萌芽状态。人工视觉解决临床上某些严重致盲眼病无有效治疗�Retinal degenerative diseases, such as retinitis pigmentosa （RP）, age-related macular degeneration （AMD）, as well as glaucoma and other diseases the main pathological basis of abnormal structure and function of retinal neurons at all levels, resulting in irreversible damage to the visual acuity, is the leading cause of irreversible blindness, and lack of effective treatment methods. With the rapid development of science and technology in recent years, gene therapy, stem cell therapy, adaptive optics technology and artificial vision to the treatment of these diseases may be provided. The completion of the human genome project and the wide application of the related sequencing technology have brought a new revolution to the nature of the disease, and also brought new impetus to the disease molecular classification, biological diagnosis and gene therapy. People＇s understanding of the eye disease is also more comprehensive, the treatment plan on the basis of gene replacement therapy,and further integrated cell factor therapy,and the treatment of light genetics, etc.. The gene therapy technology has made great progress in Department of Ophthalmology,but it can not stop some of the functions of cell apoptosis,which requires us to combine a class of genes that can save the cell apoptosis, or need to use alternative treatment, the functional cells instead of the injured cells, thereby restoring its function. Because of the unlimited proliferation and multi diffel^ntiation potential of stem cells, it has become a new hope for cell replacement therapy. The retinal stem cell transplantation, which is integrated into the retina, and differentiated into the target cells, to reconstruct the retinal function, has brought the light to the irreversible blindness. The self-renewal and multiple differentiation potential of neural stem cells, which are suitable for the cell transplantation, and the expansion of the widely used neural stem cells in vitro and in vitro culture and amplification technology, provide us with ple

关 键 词：前沿 生物技术 视网膜退行性疾病 整合医学 

分 类 号：R774.1[医药卫生—眼科]