Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study

Testing for binding or neutralizing antibodies (NAbs) to adeno-associated virus (AAV) is part of the laboratory assessment of people with hemophilia considering AAV-based gene therapy. We evaluated the natural history of NAb titers to AAV serotype 5 (AAV5) in adult males ≥18 years old with hemophili...

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Autores principales:	Robert Klamroth, Michael Recht, Nigel S. Key, Wolfgang Miesbach, Steven W. Pipe, Radoslaw Kaczmarek, Douglass Drelich, Blanca Salazar, Sandra Le Quellec, Paul E. Monahan, Nicholas Galante, Paul van der Valk, Jacqueline Tarrant
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	Elsevier 2025-09-01
Colección:	Molecular Therapy: Methods & Clinical Development
Materias:	adeno-associated virus serotype 5 hemophilia B etranacogene dezaparvovec neutralizing antibody anti-AAV5 factor IX
Acceso en línea:	http://www.sciencedirect.com/science/article/pii/S2329050125001639
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