Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC

Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC

Abstract Background Mucopolysaccharidosis IIIC (MPSIIIC) is one of four Sanfilippo diseases sharing clinical symptoms of severe cognitive decline and shortened lifespan. The missing enzyme, heparan sulfate acetyl-CoA: α-glucosaminide-N-acetyltransferase (HGSNAT), is bound to the lysosomal membrane,...

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Bibliographic Details
Main Authors: Claire O’Leary, Gabriella Forte, Nadia L. Mitchell, Amir Saam Youshani, Adam Dyer, Martin P. Wellby, Katharina N. Russell, Samantha J. Murray, Nelly Jolinon, Simon A Jones, Kevin Stacey, Daniel M. Davis, Els Henckaerts, David N. Palmer, Ian Kamaly-Asl, Brian W. Bigger
Format: Article
Language:English
Published: BMC 2023-07-01
Series:Journal of Translational Medicine
Subjects:
AAV gene therapy
Convection Enhanced Delivery
HGSNAT
Large animal
Mucopolysaccharidosis
Online Access:https://doi.org/10.1186/s12967-023-04208-1

Internet

https://doi.org/10.1186/s12967-023-04208-1

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