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Article

ABCA4 disease progression and a proposed strategy for gene therapy.

Human molecular genetics - 1 Mar 2009

Cideciyan Artur V, Swider Malgorzata, Aleman Tomas S, Tsybovsky Yaroslav, Schwartz Sharon B, Windsor Elizabeth A M, Roman Alejandro J, Sumaroka Alexander, Steinberg Janet D, Jacobson Samuel G, Stone Edwin M, Palczewski Krzysztof

Abstract excerpt

Autosomal recessive retinal diseases caused by mutations in the ABCA4 gene are being considered for gene replacement therapy. All individuals with ABCA4-disease show macular degeneration, but only some are thought to progress to retina-wide blindness. It is currently not predictable if or when sp...

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Source

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Identifiers

PMID: 19074458
DOI: 10.1093/hmg/ddn421
PMCID: PMC2640207

Genes

ABCA4

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-19074458-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

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