AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life

The hemophilias are the most common severe inherited bleeding disorders and are caused by deficiency of clotting factor (F) VIII (hemophilia A) or FIX (hemophilia B). The resultant bleeding predisposition significantly increases morbidity and mortality. The ability to improve the bleeding phenotype...

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Bibliographic Details

Published in:	Molecular Therapy: Methods & Clinical Development
Main Authors:	Bhavya S. Doshi, Benjamin J. Samelson-Jones, Timothy C. Nichols, Elizabeth P. Merricks, Joshua L. Siner, Robert A. French, Ben J. Lee, Valder R. Arruda, Mary Beth Callan
Format:	Article
Language:	English
Published:	Elsevier 2024-03-01
Subjects:	AAV gene therapy hemophilia companion animals real world efficacy
Online Access:	http://www.sciencedirect.com/science/article/pii/S2329050124000214