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Recombinant factor IX secreted by transduced human keratinocytes is biologically active
Author(s) -
Gerrard A. J.,
Austen D. E. G.,
Brownlee G. G.
Publication year - 1996
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.1365-2141.1996.d01-1923.x
Subject(s) - recombinant dna , factor ix , biological activity , haemophilia , biology , microbiology and biotechnology , chemistry , immunology , in vitro , biochemistry , gene , genetics
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.