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Article

Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2007

Mah Cathryn, Pacak Christina A, Cresawn Kerry O, Deruisseau Lara R, Germain Sean, Lewis Melissa A, Cloutier Denise A, Fuller David D, Byrne Barry J

Abstract excerpt

Pompe disease is caused by a lack of functional lysosomal acid alpha-glucosidase (GAA) and can ultimately lead to fatal hypertrophic cardiomyopathy and respiratory insufficiency. Previously, we demonstrated the ability of recombinant adeno-associated virus serotype 1 (rAAV2/1) vector to restore the therapeutic levels of cardiac and diaphragmatic GAA enzymatic activity in vivo in a mouse model of Pompe disease. We...

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Source

Identifiers

PMID: 17245350
DOI: 10.1038/sj.mt.6300100

Genes

GAA

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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