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Article

Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene.

Gene therapy - 1 Nov 2010

Koppanati B M, Li J, Reay D P, Wang B, Daood M, Zheng H, Xiao X, Watchko J F, Clemens P R

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a devastating primary muscle disease with pathological changes in skeletal muscle that are ongoing at the time of birth. Progressive deterioration in striated muscle function in affected individuals ultimately results in early death due to cardio-pulmonary failure. As affected individuals can be identified before birth by prenatal genetic testing for DMD, gene replacement...

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Source

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Identifiers

PMID: 20535217
DOI: 10.1038/gt.2010.84
PMCID: PMC2939256

Genes

DMD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene.PMID 20535217Read ↗

Systemic human <i>minidystrophin</i> gene transfer improves functions and life span of dystrophin and dystrophin/utrophin‐deficient miceSemantic proximity Read ↗

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