Research progress of gene therapy for retinitis pigmentosa
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    Abstract:

    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).

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Fang-Yuan Deng, Meng-Yu Han, Ting-Ting Deng, et al. Research progress of gene therapy for retinitis pigmentosa. Guoji Yanke Zazhi( Int Eye Sci) 2021;21(7):1205-1208

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Publication History
  • Received:
  • Revised:May 26,2021
  • Adopted:
  • Online: June 24,2021
  • Published: