Prednisolone Does Not Regulate Factor VIII Expression in Mice Receiving AAV5-hFVIII-SQ: Valoctocogene Roxaparvovec

AAV5-hFVIII-SQ (valoctocogene roxaparvovec) is an adeno-associated virus (AAV)-mediated gene therapy vector containing a B-domain-deleted human factor VIII (hFVIII-SQ) transgene. In a phase 1/2 clinical study of AAV5-hFVIII-SQ for severe hemophilia A (FVIII < 1 IU/dL), participants received predn...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Zhang, Lening, Handyside, Britta, Murphy, Ryan, Sihn, Choong-Ryoul, Xie, Lin, Vitelli, Catherine, Harmon, Danielle, Sisó, Sílvia, Liu, Su, Bullens, Sherry, Bunting, Stuart, Fong, Sylvia
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6923509/
https://ncbi.nlm.nih.gov/pubmed/31890737
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.11.007
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