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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Principais autores:	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
פורמט:	Artigo
שפה:	Inglês
יצא לאור:	Elsevier 2024-03-01
סדרה:	Molecular Therapy: Methods & Clinical Development
נושאים:	AAV gene therapy hemophilia companion animals real world efficacy
גישה מקוונת:	http://www.sciencedirect.com/science/article/pii/S2329050124000214
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