Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy

Background: Previously, we reported data from a first-in-human, dose-escalation, Phase 1 gene therapy trial demonstrating a dose-dependent increase in transgenic factor IX (FIX) expression in 10 adults with severe hemophilia B (FIX activity ≤1%). This was achieved following a single intravenous infu...

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Veröffentlicht in:	Blood 2023-11, Vol.142 (Supplement 1), p.1056-1056
Hauptverfasser:	Reiss, Ulrike M., Davidoff, Andrew M., Tuddenham, Edward G. D., Chowdary, Pratima, McIntosh, Jenny H., Riddell, Anne, Pie, Arnulfo, Batty, Paul, Calvert, Joanna C. M., Mangles, Sarah, Mahlangu, Johnny, Haley, Kristina M., Recht, Michael, Shen, Yu-Min, Halka, Kathleen G., Zhou, Junfang, Kang, Guolian, Okhomina, Victoria, Morton, Christopher L, Nathwani, Amit C.
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
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