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

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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Bibliographic Details
Main Authors: Sylvia Fong, Britta Handyside, Choong-Ryoul Sihn, Su Liu, Lening Zhang, Lin Xie, Ryan Murphy, Nicole Galicia, Bridget Yates, Wesley C. Minto, Catherine Vitelli, Danielle Harmon, Yuanbin Ru, Guoying Karen Yu, Claudia Escher, Jakob Vowinckel, Jill Woloszynek, Hassib Akeefe, Rajeev Mahimkar, Sherry Bullens, Stuart Bunting
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV5
endoplasmic reticulum
hemophilia A
gene therapy
valoctocogene roxaparvovec
molecular chaperone
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301546

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http://www.sciencedirect.com/science/article/pii/S2329050120301546

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