Improved intravenous lentiviral gene therapy based on endothelial-specific promoter-driven factor VIII expression for hemophilia A

Improved intravenous lentiviral gene therapy based on endothelial-specific promoter-driven factor VIII expression for hemophilia A

Abstract Background Hemophilia A (HA) is an X-linked monogenic disorder caused by deficiency of the factor VIII (FVIII) gene in the intrinsic coagulation cascade. The current protein replacement therapy (PRT) of HA has many limitations including short term effectiveness, high cost, and life-time tre...

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Bibliographic Details
Main Authors: Jie Gong, Rui Yang, Min Zhou, Lung-Ji Chang
Format: Article
Language:English
Published: BMC 2023-06-01
Series:Molecular Medicine
Subjects:
Lentiviral vector
Gene therapy
Hemophilia A
Factor VIII
Endothelial-specific
Online Access:https://doi.org/10.1186/s10020-023-00680-z

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