Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A

ESSENTIALS: Factor (F) VIII is an inefficiently expressed protein. Furin deletion FVIII variants were purified and characterized using in vitro and in vivo assays. These minimally modified novel FVIII variants have enhanced function. These variants provide a strategy for increasing FVIII expression...

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Detalles Bibliográficos
Autores principales: Nguyen, G. N., George, L. A., Siner, J. I., Davidson, R. J., Zander, C. B., Zheng, X. L., Arruda, V. R., Camire, R. M., Sabatino, D. E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2016
Materias:
COAGULATION
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5280213/
https://www.ncbi.nlm.nih.gov/pubmed/27749002
http://dx.doi.org/10.1111/jth.13543

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5280213/
https://www.ncbi.nlm.nih.gov/pubmed/27749002
http://dx.doi.org/10.1111/jth.13543

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