Bennett Jean, Chung Daniel C, Maguire Albert
F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Methods Enzymol. 2012;507:255-74. doi: 10.1016/B978-0-12-386509-0.00013-2.
With the recent progress in identifying disease-causing genes in humans and in animal models, there are more and more opportunities for using retinal gene transfer to learn more about retinal physiology and also to develop therapies for blinding disorders. Success in preclinical studies for one form of inherited blindness have led to testing in human clinical trials. This paves the way to consider a number of other retinal diseases as ultimate gene therapy targets in human studies. The information presented here is designed to assist scientists and clinicians to use gene transfer to probe the biology of the retina and/or to move appropriate gene-based treatment studies from the bench to the clinic.
随着近期在人类和动物模型中鉴定致病基因方面取得的进展，利用视网膜基因转移来更多地了解视网膜生理学以及开发治疗致盲疾病的疗法的机会越来越多。针对一种遗传性失明形式的临床前研究取得成功，已促使开展人体临床试验。这为在人体研究中将许多其他视网膜疾病视为最终的基因治疗靶点铺平了道路。本文提供的信息旨在帮助科学家和临床医生利用基因转移来探究视网膜生物学和/或将适当的基于基因的治疗研究从实验室推向临床。
