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Article

Recent advances in early-onset severe retinal degeneration: more than just basic research.

Trends in molecular medicine - 1 Feb 2004

Preising Markus N, Heegaard Steffen

Abstract excerpt

Successful treatment of early-onset sever retinal degeneration (EOSRD) in an animal model of the disease has provided the first proof-o-principle for retinal gene therapy of higher mammals. Currently, large sets of DNA samples are screened to identify patients with Leber's congenital amaurosis (LCA) carrying mutations in RPE65 as possible candidates for gene therapy trials. Research into EOSRD and LCA aims to...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Share to Optic Atrophy, Hereditary, Leber

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Source

Identifiers

PMID: 15106616
DOI: 10.1016/j.molmed.2003.12.002

Genes

RPE65

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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