Department of Ophthalmology & Visual Science, University of British Columbia, Eye Care Centre, Vancouver, BC, Canada.
Curr Gene Ther. 2012 Apr 1;12(2):103-15. doi: 10.2174/156652312800099607.
Ex vivo gene therapy, a technique where genetic manipulation of cells is undertaken remotely and more safely since it is outside the body, is an emerging therapeutic strategy particularly well suited to targeting a specific organ rather than for treating a whole organism. The eye and visual pathways therefore make an attractive target for this approach. With blindness still so prevalent worldwide, new approaches to treatment would also be widely applicable and a significant advance in improving quality of life. Despite being a relatively new approach, ex vivo gene therapy has already achieved significant advances in the treatment of blindness in pre-clinical trials. In particular, advances are being achieved in corneal disease, glaucoma, retinal degeneration, stroke and multiple sclerosis through genetic re-programming of cells to replace degenerate cells and through more refined neuroprotection, modulation of inflammation and replacement of deficient protein. In this review we discuss the latest developments in ex vivo gene therapy relevant to the visual pathways and highlight the challenges that need to be overcome for progress into clinical trials.
离体基因治疗是一种在体外进行细胞遗传操作的技术，由于它在体外进行，因此更加安全。它是一种新兴的治疗策略，特别适合针对特定器官，而不是治疗整个生物体。眼睛和视觉通路因此成为这种方法的一个有吸引力的目标。由于全球范围内仍然存在着如此高的失明率，新的治疗方法也将具有广泛的适用性，并在提高生活质量方面取得重大进展。尽管这是一种相对较新的方法，但离体基因治疗在临床前试验中已经在治疗失明方面取得了重大进展。特别是，通过对细胞进行遗传重编程以替代退化细胞，以及通过更精细的神经保护、炎症调节和缺失蛋白的替代，在角膜疾病、青光眼、视网膜变性、中风和多发性硬化症方面取得了进展。在这篇综述中，我们讨论了与视觉通路相关的离体基因治疗的最新进展，并强调了需要克服的挑战，以推进临床试验。
