Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

Background Gene therapy for hemophilia B (HB) consisting of a one-time, systemic, administration of adeno-associated viral (AAV) vectors encoding a functional Factor-IX gene is now approved and represents an important and long-awaited milestone. A rise in anti-AAV antibody titers follows systemic ad...

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Veröffentlicht in:Blood 2023-11, Vol.142 (Supplement 1), p.2255-2255
Hauptverfasser: McIntosh, Jenny H., Mohamed, Saira, Ireland, Lydia, Morton, Christopher L, Reiss, Ulrike M., Chowdary, Pratima, Tuddenham, Edward G. D., Davidoff, Andrew M., Nathwani, Amit C.
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Sprache:eng
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