Progression of gene therapy in retinal disease treatments

How to Cite

Dongye, M. . (2017). Progression of gene therapy in retinal disease treatments. American Journal of Translational Medicine, 1(3), 148–160. Retrieved from


Retinal diseases have long drawn scientists’ attention, and many of these diseases remain untreatable, placing affected patients at risk of suffering from lifelong blindness. The progression of DNA sequencing has helped ophthalmologists discover the altered genes in retinal diseases and paved the way for gene therapy. With their unique biological structure and immune advantages, eyes are ideal materials for gene therapy applications. Our review will summarize the different gene therapy methods applied in several retinal diseases, among which the strategy of gene compensation is widely used. The newly developed CRISPR/Cas system serves as a powerful gene editing tool and shows great potential in gene correction, which is a promising avenue in future gene therapy. (Am J Transl Med 2017. 1:148-160)