Aj. Gerrard et al., RECOMBINANT FACTOR-IX SECRETED BY TRANSDUCED HUMAN KERATINOCYTES IS BIOLOGICALLY-ACTIVE, British Journal of Haematology, 95(3), 1996, pp. 561-563
We have been investigating the use of human keratinocytes as potential
target cells for gene therapy for haemophilia B, with the aim of curi
ng haemophilia by means of factor IX secreting skin graft. Previous st
udies indicated that keratinocytes might be suitable cells, although a
potential problem was that the recombinant factor IX secreted by tran
sduced keratinocytes was found to be only 40% biologically active, We
now report, using an alternative assay to test for activity, that the
secreted factor IX appears to be almost fully active.