Recombinant factor IX secreted by transduced human keratinocytes is biologically active.
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.
Gerrard
A J
AJ
Chemical Pathology Unit, Sir William Dunn School of Pathology, University of Oxford.
Austen
D E
DE
Brownlee
G G
GG
D005164:Factor IX / Q000378:metabolism*; D015316:Genetic Therapy; D006467:Hemophilia A / Q000378:metabolism* / Q000628:therapy; D006801:Humans; D015603:Keratinocytes / Q000378:metabolism*; D011994:Recombinant Proteins
D011994:Recombinant Proteins; D005164:Factor IX