Zhonghua Yan Ke Za Zhi. 2011 Jan;47(1):83-7.

[Progress in gene therapy study of Leber congenital amaurosis].

[Article in Chinese]

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Eye Hospital, Wenzhou Medical College, Wenzhou 325027, China.

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.

PMID:: 21418933; [PubMed - indexed for MEDLINE]

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[Progress in gene therapy study of Leber congenital amaurosis].

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