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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Bibliografische gegevens

Hoofdauteurs:	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
Formaat:	Artigo
Taal:	Inglês
Gepubliceerd in:	Elsevier 2025-06-01
Reeks:	Molecular Therapy: Methods & Clinical Development
Onderwerpen:	Pompe disease AAV gene therapy muscle targeting liver detargeting immune response
Online toegang:	http://www.sciencedirect.com/science/article/pii/S2329050125000592
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