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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
Publikováno v:
American Journal of Mechanical and Industrial Engineering. 3:80
With the recent advancement in complexity of various technological products and the need for faster production, the quest for inventing new manufacturing technologies to reduce the design limitations such as the costs involved in purchasing and maint