Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

Pompe disease results from acid α-glucosidase (GAA) deficiency, and enzyme replacement therapy (ERT) with recombinant human (rh) GAA has clinical benefits, although its limitations include the short half-life of GAA and the formation of antibody responses. The present study compared the efficacy of...

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Bibliographic Details
Main Authors: Sang-oh Han, Giuseppe Ronzitti, Benjamin Arnson, Christian Leborgne, Songtao Li, Federico Mingozzi, Dwight Koeberl
Format: Article
Language:English
Published: Elsevier 2017-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
adeno-associated virus
acid α-glucosidase
acid maltase
glycogen storage disease type II
immune tolerance induction
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117300049

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http://www.sciencedirect.com/science/article/pii/S2329050117300049

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