Factor IX gene therapy for the treatment of hemophilia a complicated with inhibitory antibodies

The treatment of patients with inhibitory antibodies remains a major challenge in hemophilia therapy. Here, we pursue a gene transfer strategy using FIX variants which do not require FVIII. In initial variant screening experiments, we combined three to five amino acid substitutions which led to the...

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Veröffentlicht in:	Human gene therapy 2009-11, Vol.20 (11), p.1474-1474
Hauptverfasser:	Milanov, P, Abriss, D, Tonn, T, Seifried, E, Schuettrumpf, J
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
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