Article
Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.
Blood - 1 Nov 2004
Waddington Simon N, Nivsarkar Megha S, Mistry Ajay R, Buckley Suzanne M K, Kemball-Cook Geoffrey, Mosley Karen L, Mitrophanous Kyriacos, Radcliffe Pippa, Holder Maxine V, Brittan Mairi, Georgiadis Anastasios, Al-Allaf Faisal, Bigger Brian W, Gregory Lisa G, Cook H Terence, Ali Robin R, Thrasher Adrian, Tuddenham Edward G D, Themis Mike, Coutelle Charles
Abstract excerpt
Hemophilia B, also known as Christmas disease, arises from mutations in the factor IX (F9) gene. Its treatment in humans, by recombinant protein substitution, is expensive, thus limiting its application to intermittent treatment in bleeding episodes and prophylaxis during surgery; development of inhibitory antibodies is an associated hazard. This study demonstrates permanent therapeutic correction of his disease...
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