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Research progress of gene therapy for retinitis pigmentosa / 国际眼科杂志(Guoji Yanke Zazhi)
International Eye Science ; (12): 1205-1208, 2021.
Artigo em Chinês | WPRIM | ID: wpr-877385
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).

Texto completo: DisponíveL Índice: WPRIM (Pacífico Ocidental) Idioma: Chinês Revista: International Eye Science Ano de publicação: 2021 Tipo de documento: Artigo

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Texto completo: DisponíveL Índice: WPRIM (Pacífico Ocidental) Idioma: Chinês Revista: International Eye Science Ano de publicação: 2021 Tipo de documento: Artigo