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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...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Zolotukhin, Irene, Markusic, David M, Palaschak, Brett, Hoffman, Brad E, Srikanthan, Meera A, Herzog, Roland W
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5040172/
https://ncbi.nlm.nih.gov/pubmed/27738644
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2016.63
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