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Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

Currently, individuals with pre-existing neutralizing antibodies (NABs) against adeno-associated virus (AAV) above titer of 5 are excluded from systemic AAV-based clinical trials. In this study we explored the impact of pre-existing anti-AAV5 NABs on the efficacy of AAV5-based gene therapy. AMT-060...

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Detalles Bibliográficos
Autores principales:	Majowicz, Anna, Nijmeijer, Bart, Lampen, Margit H., Spronck, Lisa, de Haan, Martin, Petry, Harald, van Deventer, Sander J., Meyer, Christian, Tangelder, Marco, Ferreira, Valerie
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6586596/ https://www.ncbi.nlm.nih.gov/pubmed/31276009 http://dx.doi.org/10.1016/j.omtm.2019.05.009

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