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

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, therefor...

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Detalles Bibliográficos
Autores principales: O’Leary, Claire, Forte, Gabriella, Mitchell, Nadia L., Youshani, Amir Saam, Dyer, Adam, Wellby, Martin P., Russell, Katharina N., Murray, Samantha J., Jolinon, Nelly, Jones, Simon A, Stacey, Kevin, Davis, Daniel M., Henckaerts, Els, Palmer, David N., Kamaly-Asl, Ian, Bigger, Brian W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10320977/
https://www.ncbi.nlm.nih.gov/pubmed/37407981
http://dx.doi.org/10.1186/s12967-023-04208-1

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