Research progress of gene therapy for retinitis pigmentosa

Research progress of gene therapy for retinitis pigmentosa

Retinitis pigmentosa(RP)is a hereditary retinal disease characterized by degeneration of retina rods and cones photoreceptor cells and degeneration of retinal pigment epithelial cells. The age of onset and progression of RP are genetically related and influenced by the environment. Gene therapy uses...

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Bibliographic Details
Main Authors: Fang-Yuan Deng, Meng-Yu Han, Ting-Ting Deng, Ming Jin
Format: Article
Language:English
Published: Press of International Journal of Ophthalmology (IJO PRESS) 2021-07-01
Series:Guoji Yanke Zazhi
Subjects:
retinitis pigmentosa
gene therapy
research progress
review
Online Access:http://ies.ijo.cn/cn_publish/2021/7/202107015.pdf
Description
Summary:Retinitis pigmentosa(RP)is a hereditary retinal disease characterized by degeneration of retina rods and cones photoreceptor cells and degeneration of retinal pigment epithelial cells. The age of onset and progression of RP are genetically related and influenced by the environment. Gene therapy uses vectors in delivering therapeutic genes to genetically modify target cells, so as to correct or replace the disease-causing RP genes. This article introduces the research progress of vectors in RP gene therapy, and review the efficacy and safety of gene therapy on five common genotypes(RHO, PDE6B, MERTK, RLBP1, RPGR).
ISSN:1672-5123

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