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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Hlavní autoři:	Choong-Ryoul Sihn, Britta Handyside, Su Liu, Lening Zhang, Ryan Murphy, Bridget Yates, Lin Xie, Richard Torres, Chris B. Russell, Charles A. O'Neill, Erno Pungor, Stuart Bunting, Sylvia Fong
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2022-03-01
Edice:	Molecular Therapy: Methods & Clinical Development
Témata:	AAV vectors AAV genome processing oversized AAV vectors circular episomes hepatocyte-directed AAV gene transfer AAV5-hFVIII-SQ
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S2329050121001923
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