Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency

One important limitation for achieving therapeutic expression of human factor VIII (FVIII) in hemophilia A gene therapy is inefficient secretion of the FVIII protein. Substitution of five amino acids in the A1 domain of human FVIII with the corresponding porcine FVIII residues generated a secretion-...

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Detalles Bibliográficos
Autores principales: Cao, Wenjing, Dong, Biao, Horling, Franziska, Firrman, Jenni A., Lengler, Johannes, Klugmann, Matthias, de la Rosa, Maurus, Wu, Wenman, Wang, Qizhao, Wei, Hongying, Moore, Andrea R., Roberts, Sean A., Booth, Carmen J., Hoellriegl, Werner, Li, Dong, Konkle, Barbara, Miao, Carol, Reipert, Birgit M., Scheiflinger, Friedrich, Rottensteiner, Hanspeter, Xiao, Weidong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7708868/
https://www.ncbi.nlm.nih.gov/pubmed/33313336
http://dx.doi.org/10.1016/j.omtm.2020.10.013

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