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Transduction of modified factor VIII gene improves lentiviral gene therapy efficacy for hemophilia A

Hemophilia A (HA) is a bleeding disorder caused by deficiency of the coagulation factor VIII (F8). F8 replacement is standard of care, whereas gene therapy (F8 gene) for HA is an attractive investigational approach. However, the large size of the F8 gene and the immunogenicity of the product present...

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Detalles Bibliográficos
Autores principales:	Gong, Jie, Chung, Tsai-Hua, Zheng, Jie, Zheng, Huyong, Chang, Lung-Ji
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society for Biochemistry and Molecular Biology 2021
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8649223/ https://www.ncbi.nlm.nih.gov/pubmed/34774524 http://dx.doi.org/10.1016/j.jbc.2021.101397

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