Article
Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX.
Gene therapy - 1 Jan 2006
Bigger B W, Siapati E K, Mistry A, Waddington S N, Nivsarkar M S, Jacobs L, Perrett R, Holder M V, Ridler C, Kemball-Cook G, Ali R R, Forbes S J, Coutelle C, Wright N, Alison M, Thrasher A J, Bonnet D, Themis M
Abstract excerpt
Immune responses against an introduced transgenic protein are a potential risk in many gene replacement strategies to treat genetic disease. We have developed a gene delivery approach for hemophilia B based on lentiviral expression of human factor IX in purified hematopoietic stem cells. In both normal C57Bl/6J and hemophilic 129/Sv recipient mice, we observed the production of therapeutic levels of human factor...
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