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Translational Data from Adeno-Associated Virus-Mediated Gene Therapy of Hemophilia B in Dogs

12 Feb 2015

Abstract excerpt

Preclinical testing of new therapeutic strategies in relevant animal models is an essential part of drug development. The choice of animal models of disease that are used in these studies is driven by the strength of the translational data for informing about safety, efficacy, and success or failure of human clinical trials. Hemophilia B is a monogenic, X-linked, inherited bleeding disorder that results from...

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PMID: 25675273
DOI: 10.1089/humc.2014.153

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