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

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 treatment r...

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Detalles Bibliográficos
Autores principales:	Gong, Jie, Yang, Rui, Zhou, Min, Chang, Lung-Ji
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2023
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10262495/ https://www.ncbi.nlm.nih.gov/pubmed/37308845 http://dx.doi.org/10.1186/s10020-023-00680-z

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