title: Recombinant factor IX secreted by transduced human keratinocytes is biologically active.
abstract: We have been investigating the use of human keratinocytes as potential target cells for gene therapy for haemophilia B, with the aim of curing haemophilia by means of a factor IX secreting skin graft. Previous studies indicated that keratinocytes might be suitable cells, although a potential problem was that the recombinant factor IX secreted by transduced 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.
authors.affiliation: Chemical Pathology Unit, Sir William Dunn School of Pathology, University of Oxford.
authors.forename: A J
authors.initials: AJ
authors.lastname: Gerrard
authors.forename: D E
authors.initials: DE
authors.lastname: Austen
authors.forename: G G
authors.initials: GG
authors.lastname: Brownlee
mesh_terms: D005164:Factor IX / Q000378:metabolism*; D015316:Genetic Therapy; D006467:Hemophilia A / Q000378:metabolism* / Q000628:therapy; D006801:Humans; D015603:Keratinocytes / Q000378:metabolism*; D011994:Recombinant Proteins
chemical_list: D011994:Recombinant Proteins; D005164:Factor IX