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Article

The effect of human gene therapy for RPE65-associated Leber's congenital amaurosis on visual function: a systematic review and meta-analysis.

Orphanet journal of rare diseases - 14 Feb 2020

Wang Xue, Yu Chaofeng, Tzekov Radouil T, Zhu Yihua, Li Wensheng

Abstract excerpt

BACKGROUND: RPE65-associated LCA (RPE65-LCA) is an inherited retinal degeneration caused by the mutations of RPE65 gene and gene therapy has been developed to be a promising treatment. This study aims to evaluate the association between changes in visual function and application of gene therapy in patients with RPE65-LCA. METHODS: Several databases (PubMed, Cochrane Library, and Web of Science) were searched for...

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Source

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Identifiers

PMID: 32059734
DOI: 10.1186/s13023-020-1304-1
PMCID: PMC7023818

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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