Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity-Reference-Cited by-同舟云学术

Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity

Published:2021-12 Issue:1 Volume:14 Page:
ISSN:1757-4676
Container-title:EMBO Molecular Medicine
language:en
Short-container-title:EMBO Mol Med

Author:

Eggers Michelle¹^ORCID,Vannoy Charles H¹^ORCID,Huang Jianyong¹,Purushothaman Pravinkumar¹,Brassard Jacqueline²^ORCID,Fonck Carlos¹,Meng Hui³,Prom Mariah J³,Lawlor Michael W³,Cunningham Justine¹,Sadhu Chanchal¹,Mavilio Fulvio¹⁴^ORCID

Affiliation:

1. Nonclinical, Pharmacology/Toxicology Audentes Therapeutics San Francisco CA USA

2. Jacqueline Brassard Toxicologic Pathology Consulting Tustin CA USA

3. Department of Pathology and Neuroscience Research Center Medical College of Wisconsin Milwaukee WI USA

4. Department of Life Sciences University of Modena and Reggio Emilia Modena Italy

Funder

Audentes Therapeutics

Publisher

EMBO

Subject

Molecular Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.15252/emmm.202113968

Reference36 articles.

1. Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease

2. Gene Therapy for Pompe Disease: The Time is now

3. Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

4. Induction of antigen-specific CD4+ T-cell anergy and deletion by in vivo viral gene transfer

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

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