The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion
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DOI: 10.1182/blood-2019-126489
Abstract: Introduction. Adeno-associated virus (AAV)-based factor VIII (FVIII) gene therapy holds great promise to provide clinical benefit in patients with hemophilia A. However, very high doses are currently needed to achieve therapeutic factor levels and the… read more here.
Keywords: baxalta innovations; fviii; gmbh takeda; innovations gmbh ... See more keywords