Oral gene therapy for hemophilia B using chitosan‐formulated FIX mutants

Summary Background Oral gene delivery of non‐viral vectors is an attractive strategy to achieve transgene expression. Although expected efficacy from non‐viral delivery systems is relatively low, repeated vector administration is possible and may help to obtain durable transgene expression in a ther...

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Veröffentlicht in:	Journal of thrombosis and haemostasis 2014-06, Vol.12 (6), p.932-942
Hauptverfasser:	Quade‐Lyssy, P., Milanov, P., Abriss, D., Ungerer, C., Königs, C., Seifried, E., Schüttrumpf, J.
Format:	Artikel
Sprache:	eng
Schlagworte:	Administration, Oral Animals chitosan Chitosan - administration & dosage collagen type IV Disease Models, Animal factor IX Factor IX - administration & dosage Factor IX - biosynthesis Factor IX - genetics Feasibility Studies gene therapy Genetic Therapy - methods Genotype HEK293 Cells hemophilia B Hemophilia B - blood Hemophilia B - genetics Hemophilia B - therapy Hemostasis Humans Intestine, Small - metabolism Liver - metabolism Mice, Inbred C57BL Mice, Knockout Mutagenesis, Site-Directed Mutation Nanoparticles Phenotype Time Factors Transfection
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