Ramachandran Pavitra S, Song Ji Yun, Bennett Jean
J Clin Invest. 2015 Apr;125(4):1390-2. doi: 10.1172/JCI80821. Epub 2015 Mar 23.
The use of gene therapy for blinding disease shows growing promise; however, due to an ever-expanding list of disease-causing genes and mutations, the identification of a generic gene-based treatment is urgently needed. In many forms of degenerative retinal disease, there may be a window of opportunity to preserve daylight vision, as the cone photoreceptors degenerate more slowly than do the rods. In this issue of the JCI, Venkatesh et al. and Xiong et al. exploit two different pathways to promote cone cell survival and preserve vision in murine retinal degeneration models. These studies provide hope for developing a universal reagent to treat many different blinding disorders.
基因疗法用于治疗致盲疾病显示出越来越大的前景;然而,由于致病基因和突变的种类不断增加,迫切需要找到一种基于基因的通用治疗方法。在许多形式的视网膜退行性疾病中,可能存在保留明视觉的机会窗口,因为视锥光感受器的退化比视杆光感受器更慢。在本期《临床研究杂志》中,文卡特什等人和熊等人利用两种不同的途径来促进小鼠视网膜变性模型中的视锥细胞存活并保留视力。这些研究为开发一种治疗多种不同致盲疾病的通用试剂带来了希望。