Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease-Reference-Cited by-同舟云学术

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Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

Published:2016-01 Issue:1 Volume:27 Page:43-59
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Doerfler Phillip A.¹,Todd Adrian G.¹,Clément Nathalie¹,Falk Darin J.¹,Nayak Sushrusha²,Herzog Roland W.³,Byrne Barry J.¹

Affiliation:

1. Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, Florida

2. Department of Medicine, Center for Infectious Medicine, Karolinska Institute, Stockholm, Sweden

3. Division of Cellular & Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, Florida.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2015.103

Reference92 articles.

1. Immune responses to AAV vectors: overcoming barriers to successful gene therapy

2. Pompe's disease

3. Pompe disease: Design, methodology, and early findings from the Pompe Registry

4. Recombinant human acid ??-glucosidase enzyme therapy for infantile glycogen storage disease type II: Results of a phase I/II clinical trial

5. Glycogen storage disease types I and II: Treatment updates

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1. Neurological glycogen storage diseases and emerging therapeutics;Neurotherapeutics;2024-09

2. Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy;Molecular Diagnosis & Therapy;2024-08-12

3. Restoring immune balance with Tregitopes: A new approach to treating immunological disorders;Biomedicine & Pharmacotherapy;2024-08

4. Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver;JCI Insight;2024-05-16

5. AAV-mediated delivery of secreted acid α-glucosidase with enhanced uptake corrects neuromuscular pathology in Pompe mice;JCI Insight;2023-08-22

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