Zobrazeno 1 - 1
of 1
pro vyhledávání: '"Stephen Vorobiov"'
AAV gene replacement therapy for treating MPS IIIC: Facilitating bystander effects via EV‐mRNA cargo
Autor:
Tierra A. Bobo, Michael Robinson, Christopher Tofade, Marina Sokolski‐Papkov, Peter Nichols, Stephen Vorobiov, Haiyan Fu
Publikováno v:
Journal of Extracellular Vesicles, Vol 13, Iss 7, Pp n/a-n/a (2024)
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 I
Externí odkaz:
https://doaj.org/article/f3b1a7a5a82f4abd9c55af1cc1da434d
