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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Hlavní autoři:	Irene Zolotukhin, David M Markusic, Brett Palaschak, Brad E Hoffman, Meera A Srikanthan, Roland W Herzog
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2016-01-01
Edice:	Molecular Therapy: Methods & Clinical Development
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S2329050116301917
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