AAV gene replacement therapy for treating MPS IIIC: Facilitating bystander effects via EV‐mRNA cargo

AAV gene replacement therapy for treating MPS IIIC: Facilitating bystander effects via EV‐mRNA cargo

Abstract MPS IIIC is a lysosomal storage disease caused by mutations in heparan‐α‐glucosaminide N‐acetyltransferase (HGSNAT), for which no treatment is available. Because HGSNAT is a trans‐lysosomal‐membrane protein, gene therapy for MPS IIIC needs to transduce as many cells as possible for maximal...

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Bibliographic Details
Main Authors: Tierra A. Bobo, Michael Robinson, Christopher Tofade, Marina Sokolski‐Papkov, Peter Nichols, Stephen Vorobiov, Haiyan Fu
Format: Article
Language:English
Published: Wiley 2024-07-01
Series:Journal of Extracellular Vesicles
Subjects:
adeno‐associated virus (AAV)
blood‐brain‐barrier
bystander effects/cross‐correction
EV‐mRNA cargo
lysosomal storage diseases
mucopolysaccharidosis type IIIC (MPS IIIC)
Online Access:https://doi.org/10.1002/jev2.12464

Internet

https://doi.org/10.1002/jev2.12464

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