Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia

Adeno-associated virus (AAV) based gene therapy has demonstrated effective disease control in hemophilia. However, pre-existing immunity from wild-type AAV exposure impacts gene therapy eligibility. The aim of this multicenter epidemiologic study was to determine the prevalence and persistence of pr...

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Veröffentlicht in:Gene therapy 2024-05, Vol.31 (5-6), p.273-284
Hauptverfasser: Pabinger, Ingrid, Ayash-Rashkovsky, Mila, Escobar, Miguel, Konkle, Barbara A., Mingot-Castellano, María Eva, Mullins, Eric S., Negrier, Claude, Pan, Luying, Rajavel, Kavitha, Yan, Brian, Chapin, John
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Sprache:eng
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