Bennett J
FM Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, PA 19104-6069, USA.
Curr Opin Mol Ther. 2000 Aug;2(4):420-5.
Retinitis pigmentosa (RP) is a group of retinal degenerative diseases in which there is a slow and progressive loss of photoreceptors. There is no cure for RP and photoreceptor loss leads ultimately to blindness. There has been tremendous progress in the last decade in delineating the molecular basis of RP. Simultaneously, gene transfer experiments have demonstrated that it is possible to deliver transgenes to the retina in vivo in a stable and efficient fashion with minimal toxicity. Proof-of-principle for gene therapy for RP has been established in a number of different animal models. While much more progress needs to be made before moving from the laboratory to the clinic, gene therapy now holds much promise for slowing or even preventing blindness due to RP.
视网膜色素变性（RP）是一组视网膜退行性疾病，其中光感受器会缓慢且渐进性丧失。目前尚无治愈RP的方法，光感受器丧失最终会导致失明。在过去十年中，在阐明RP的分子基础方面取得了巨大进展。同时，基因转移实验表明，以稳定、高效且毒性最小的方式在体内将转基因递送至视网膜是可行的。在许多不同的动物模型中已经确立了RP基因治疗的原理证明。虽然在从实验室走向临床之前还需要取得更多进展，但基因治疗现在对于减缓甚至预防由RP导致的失明具有很大的前景。
