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New and improved AAVenues: current status of hemophilia B gene therapy

2 Nov 2015

Abstract excerpt

INTRODUCTION: Hemophilia B is a sex linked, monogenic, coagulation disorder caused by a deficiency in functional factor IX protein. Gene therapy for this disorder via systemic administration of an adeno-associated virus (AAV) encoding an optimized factor IX construct has shown considerable success, ameliorating the bleeding phenotype in a number of patients. However challenges to sustained curative gene transfer...

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PMID: 26524468
DOI: 10.1517/14712598.2015.1106475

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