Advances in gene therapy for retinitis pigmentosa
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Graphical Abstract
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Abstract
Retinitis pigmentosa is a group of inherited retinal diseases, which leads to progressive blindness and characterized by photoreceptor and pigment epithelial cells degeneration and apoptosis. There is no known cure for RP and the treatment aims at relieving symptoms currently. Gene sequencing brings development of gene therapy. In gene therapy, exogenous normal gene is introduced into target cells to modify or replace pathogenic gene through gene vectors, which include viral gene vector and nonviral gene vector. At present, gene therapy mainly includes gene replacement, ribozyme therapy, RNA interference and inhibition of apoptosis treatment. This article mainly reviews the current advances of gene therapy in treatment of retinitis pigmentosa.
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