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Current avenues of gene therapy in Pompe disease

19 Jul 2023

Abstract excerpt

PURPOSE OF REVIEW: Pompe disease is a rare, inherited, devastating condition that causes progressive weakness, cardiomyopathy and neuromotor disease due to the accumulation of glycogen in striated and smooth muscle, as well as neurons. While enzyme replacement therapy has dramatically changed the outcome of patients with the disease, this strategy has several limitations. Gene therapy in Pompe disease constitutes...

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PMID: 37639402
DOI: 10.1097/wco.0000000000001187

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