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Article

DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice.

The Journal of clinical investigation - 1 Sept 2012

Han Zongchao, Conley Shannon M, Makkia Rasha S, Cooper Mark J, Naash Muna I

Abstract excerpt

Mutations in the photoreceptor-specific flippase ABCA4 are associated with Stargardt disease and many other forms of retinal degeneration that currently lack curative therapies. Gene replacement is a logical strategy for ABCA4-associated disease, particularly given the current success of traditional viral-mediated gene delivery, such as with adeno-associated viral (AAV) vectors. However, the large size of the...

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Source

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Identifiers

PMID: 22886305
DOI: 10.1172/JCI64833
PMCID: PMC3428101

Genes

ABCA4

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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