Article
Codon optimization of human factor VIII cDNAs leads to high-level expression.
Blood - 20 Jan 2011
Ward Natalie J, Buckley Suzanne M K, Waddington Simon N, Vandendriessche Thierry, Chuah Marinee K L, Nathwani Amit C, McIntosh Jenny, Tuddenham Edward G D, Kinnon Christine, Thrasher Adrian J, McVey John H
Abstract excerpt
Gene therapy for hemophilia A would be facilitated by development of smaller expression cassettes encoding factor VIII (FVIII), which demonstrate improved biosynthesis and/or enhanced biologic properties. B domain deleted (BDD) FVIII retains full procoagulant function and is expressed at higher levels than wild-type FVIII. However, a partial BDD FVIII, leaving an N-terminal 226 amino acid stretch (N6), increases...
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