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

Published 2017 View Full Article

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Title
Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction
Authors
Keywords
gene therapy, adeno-associated virus, acid α-glucosidase, acid maltase, glycogen storage disease type II, immune tolerance induction
Journal
Molecular Therapy-Methods & Clinical Development
Volume 4, Issue -, Pages 126-136
Publisher
Elsevier BV
Online
2017-01-11
DOI
10.1016/j.omtm.2016.12.010

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