A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease

A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease

Pompe disease is a glycogen storage disorder caused by mutations in the acid α-glucosidase (GAA) gene, leading to reduced GAA activity and glycogen accumulation in heart and skeletal muscles. Enzyme replacement therapy with recombinant GAA, the standard of care for Pompe disease, is limited by poor...

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Bibliographic Details
Main Authors: P. Sellier, F. Collaud, Y. Krimi Benchekroun, V. Jimenez, X. Leon, N. Daniele, Q.H. Pham, J. El Andari, T. VandenDriessche, M.K. Chuah, D. Grimm, F. Bosch, F. Mingozzi, G. Ronzitti
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Pompe disease
AAV gene therapy
muscle targeting
liver detargeting
immune response
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125000592
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