Bainbridge J W, Stephens C, Parsley K, Demaison C, Halfyard A, Thrasher A J, Ali R R
Institute of Ophthalmology, University College London, UK.
Gene Ther. 2001 Nov;8(21):1665-8. doi: 10.1038/sj.gt.3301574.
We have evaluated the transduction profiles of an HIV-based lentiviral vector delivered regionally to ocular tissues in vivo. Following subretinal injection, a green fluorescent protein (GFP) reporter gene was efficiently and stably expressed in retinal pigment epithelial (RPE) cells. Limited transduction of adjacent photoreceptors occurred in newborn mice, but was inefficient in adult animals. Injection of the vector into the anterior chamber resulted in efficient and stable transduction of corneal endothelial cells. Efficient in vivo gene transfer into cells of the corneal endothelium and retinal pigment epithelium by lentiviral vectors may therefore offer a valuable approach to the treatment of disorders of the cornea and outer retina.
我们评估了一种基于HIV的慢病毒载体在体内局部递送至眼组织后的转导情况。视网膜下注射后，绿色荧光蛋白（GFP）报告基因在视网膜色素上皮（RPE）细胞中高效且稳定地表达。在新生小鼠中，相邻光感受器发生了有限的转导，但在成年动物中效率较低。将载体注入前房可导致角膜内皮细胞的高效稳定转导。因此，慢病毒载体在体内将基因有效转移至角膜内皮细胞和视网膜色素上皮细胞，可能为治疗角膜和视网膜外层疾病提供一种有价值的方法。
