Article

Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease

2024-12-16

Abstract excerpt

<h4>ABSTRACT</h4> Absence of functional acid-α-glucosidase (GAA) leads to early-onset Pompe disease with cardiorespiratory and neuromuscular failure. A novel Pompe rat model ( Gaa -/- ) was used to test the hypothesis that neonatal gene therapy with adeno-associated virus serotype 9 (AAV9) restores cardiorespiratory neuromuscular function across the lifespan. Temporal vein administration of AAV9-DES-GAA or sham...

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Identifiers and source

Literature Corpus work: b3321241-65d6-5091-b517-8d56b6f311eb
DOI: 10.1101/2024.12.10.627800

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