Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter

Adeno-associated virus 5 (AAV5)-human factor VIII-SQ (hFVIII-SQ; valoctocogene roxaparvovec) is an AAV-mediated product under evaluation for treatment of severe hemophilia A, which contains a B-domain-deleted hFVIII (hFVIII-SQ) transgene and a hybrid liver-specific promotor (HLP). To increase FVIII-...

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Publicado no:Mol Ther Methods Clin Dev
Main Authors: Fong, Sylvia, Handyside, Britta, Sihn, Choong-Ryoul, Liu, Su, Zhang, Lening, Xie, Lin, Murphy, Ryan, Galicia, Nicole, Yates, Bridget, Minto, Wesley C., Vitelli, Catherine, Harmon, Danielle, Ru, Yuanbin, Yu, Guoying Karen, Escher, Claudia, Vowinckel, Jakob, Woloszynek, Jill, Akeefe, Hassib, Mahimkar, Rajeev, Bullens, Sherry, Bunting, Stuart
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7397702/
https://ncbi.nlm.nih.gov/pubmed/32775496
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.07.005
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