Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers

Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus serotype 5 (AAV5)-based gene therapy vector containing a B-domain-deleted human coagulation factor VIII (hFVIII) gene controlled by a liver-selective promoter. AAV5-hFVIII-SQ is currently under clinical investigation as a treat...

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Detalles Bibliográficos
Autores principales: Sihn, Choong-Ryoul, Handyside, Britta, Liu, Su, Zhang, Lening, Murphy, Ryan, Yates, Bridget, Xie, Lin, Torres, Richard, Russell, Chris B., O'Neill, Charles A., Pungor, Erno, Bunting, Stuart, Fong, Sylvia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8749450/
https://www.ncbi.nlm.nih.gov/pubmed/35036471
http://dx.doi.org/10.1016/j.omtm.2021.12.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8749450/
https://www.ncbi.nlm.nih.gov/pubmed/35036471
http://dx.doi.org/10.1016/j.omtm.2021.12.004

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