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

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

Abstract Pompe disease is a severe disorder caused by loss of acid α‐glucosidase (GAA), leading to glycogen accumulation in tissues and neuromuscular and cardiac dysfunction. Enzyme replacement therapy is the only available treatment. AT845 is an adeno‐associated viral vector designed to express hum...

Full description

Bibliographic Details
Main Authors: Michelle Eggers, Charles H Vannoy, Jianyong Huang, Pravinkumar Purushothaman, Jacqueline Brassard, Carlos Fonck, Hui Meng, Mariah J Prom, Michael W Lawlor, Justine Cunningham, Chanchal Sadhu, Fulvio Mavilio
Format: Article
Language:English
Published: Springer Nature 2022-01-01
Series:EMBO Molecular Medicine
Subjects:
adeno‐associated virus
glycogen accumulation
heart inflammation
lysosomal storage disease
xenogeneic immune response
Online Access:https://doi.org/10.15252/emmm.202113968

Internet

https://doi.org/10.15252/emmm.202113968

Similar Items