Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease.
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bioRxiv [Preprint]. 2024 Dec 16:2024.12.10.627800. doi: 10.1101/2024.12.10.627800.
bioRxiv. 2024.
PMID: 39763722
Free PMC article.
Preprint.