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Stem Cell Based Gene Therapy for Pompe's Disease

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Title Stem Cell Based Gene Therapy for Pompe's Disease
Period 09 / 2008 - 10 / 2013
Status Completed
Dissertation Yes
Research number OND1336212
Data Supplier NWO

Abstract

Directed at a single therapeutic intervention for a-glucosidase deficiency (Pompe's disease), the project will evaluate efficacy and safety of lentiviral vector mediated transgenesis of hematopoietic stem cells as a new paradigm for the treatment of lysosomal enzyme deficiencies.

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Supervisor Prof.dr. A.G. Vulto
Supervisor Prof.dr. G. Wagemaker
Co-supervisor Dr. A.J.J. Reuser
Doctoral/PhD student Dr. M. Stok

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