Circulating human factor IX produced in keratin promoter transgenic mice: a feasibility study for gene therapy of haemophilia B

It has previously been suggested that keratinocytes might provide a suitable target cell for delivery of factor IX to the systemic circulation for gene therapy of haemophilia B. Here, an investigation of the use of cellular gene promoters specific for keratinocytes was undertaken to examine whether...

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Veröffentlicht in:	Human molecular genetics 1995-06, Vol.4 (6), p.993-999
Hauptverfasser:	Alexander, M.Y., Bidichandani, S.I., Cousins, F.M., Robinson, C.J.M., Duffie, E., Akhurst, R.J.
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Base Sequence Biological and medical sciences coagulation factor IX DNA Epidermis - metabolism Factor IX - genetics Factor IX - metabolism factor IX deficiency Feasibility Studies Female Gene Expression gene therapy Genetic Therapy Hematologic and hematopoietic diseases Hemophilia B - therapy Humans keratin 10 keratinocytes Keratinocytes - metabolism Keratins - genetics Male Medical sciences Mice Mice, Inbred C57BL Mice, Inbred CBA Mice, Transgenic Molecular Sequence Data Platelet diseases and coagulopathies Promoter Regions, Genetic Recombinant Fusion Proteins - genetics Recombinant Fusion Proteins - metabolism transgenic mice
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