title: [Progress in gene therapy study of Leber congenital amaurosis].
abstract: Leber congenital amaurosis (LCA) is an early onset retinal dystrophy that causes severe visual impairment. With the development of molecular genetics and the therapeutic gene replacement technology, the adeno-associated viral (AAV) vector-mediated gene therapy for LCA achieved encouraging progress in the past decade. The success of the Phase I clinical trials of human RPE65 gene therapy for LCA II patients makes it a pioneer in the field of retinal gene therapy and brings light to the cure of other hereditary retinopathy. This article briefly reviews the recent developments in the preclinical animal experiments and Phase I clinical trials for LCA.
authors.affiliation: Eye Hospital, Wenzhou Medical College, Wenzhou 325027, China.
authors.forename: Shan-Shan
authors.initials: SS
authors.lastname: Pan
authors.forename: Qin-Xiang
authors.initials: QX
authors.lastname: Zheng
authors.forename: Wen-Sheng
authors.initials: WS
authors.lastname: Li
authors.forename: Ji-Jing
authors.initials: JJ
authors.lastname: Pang
mesh_terms: D017321:Clinical Trials, Phase I as Topic; D015316:Genetic Therapy; D006801:Humans; D057130:Leber Congenital Amaurosis / Q000628:therapy*