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Potential for cellular stress response to hepatic factor VIII expression from AAV vector
Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively. Gene therapy for hemophilia with adeno-associated virus vectors has shown efficacy in hemophilia B patients. Although hemophilia A patients are more...
Autores principales: | Zolotukhin, Irene, Markusic, David M, Palaschak, Brett, Hoffman, Brad E, Srikanthan, Meera A, Herzog, Roland W |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2016
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5040172/ https://www.ncbi.nlm.nih.gov/pubmed/27738644 http://dx.doi.org/10.1038/mtm.2016.63 |
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