Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

Pompe disease is an autosomal recessive lysosomal storage disorder characterized by progressive muscle weakness. The disease is caused by mutations in the acid α-glucosidase (GAA) gene. Despite the currently available enzyme replacement therapy (ERT), roughly half of the infants with Pompe disease d...

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Bibliographic Details
Main Authors: Merel Stok, Helen de Boer, Marshall W. Huston, Edwin H. Jacobs, Onno Roovers, Trudi P. Visser, Holger Jahr, Dirk J. Duncker, Elza D. van Deel, Arnold J.J. Reuser, Niek P. van Til, Gerard Wagemaker
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
murine Pompe disease
acid α-glucosidase
lentiviral vector
hematopoietic stem cell transplantation
skeletal muscle
central nervous system
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300838

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