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Article

Gene therapy for duchenne muscular dystrophy: expectations and challenges.

Archives of neurology - 1 Sept 2007

Rodino-Klapac Louise R, Chicoine Louis G, Kaspar Brian K, Mendell Jerry R

Abstract excerpt

Duchenne muscular dystrophy is a debilitating X-linked disease with limited treatment options. We examined the possibility of moving forward with gene therapy, an approach that demonstrates promise for treating Duchenne muscular dystrophy. Gene therapy is not limited to replacement of defective g...

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Identifiers

PMID: 17846262
DOI: 10.1001/archneur.64.9.1236

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-17846262-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

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

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