Long-Term Functional Correction of Pompe Disease and Increased α-Glucosidase Expression after Gene Therapy with Novel Combinations of Muscle-Targeted Transcriptional <i>Cis</i> -Regulatory Elements

Quang Hong Pham(Vrije Universiteit Brussel), Thierry VandenDriessche(Vrije Universiteit Brussel)

Human Gene Therapy

September 30, 2025

10.1177/10430342251359989

Cited by 2

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