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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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Publicado no: | Mol Ther Methods Clin Dev |
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Main Authors: | , , , , , |
Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
Nature Publishing Group
2016
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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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