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Publication Detail
Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX
  • Publication Type:
    Journal article
  • Publication Sub Type:
  • Authors:
    Bigger BW, Siapati EK, Mistry A, Waddington SN, Nivsarkar MS, Jacobs L, Perrett R, Holder MV, Ridler C, Kemball-Cook G, Ali RR, Forbes SJ, Coutelle C, Wright N, Alison M, Thrasher AJ, Bonnet D, Themis M
  • Publication date:
  • Pagination:
    117, 126
  • Journal:
    Gene Therapy
  • Volume:
  • Issue:
  • Print ISSN:
  • Keywords:
    13, 15, 2005, A, AND, ANTIGEN, ARTICLE, BOTH, cell, CELLS, Delivery, DEVELOPED, DISEASE, EXPRESSION, FACTOR IX, FOR, GENE, Genetic, GENETIC DISEASE, Hematopoietic Stem Cells, HEMOPHILIA, Hemophilia B, Human, IM, immune response, IMMUNE RESPONSES, IMMUNE-RESPONSES, IS, JOURNAL, LA, LEVEL, LONDON, LONG-TERM, Low, medicine, MICE, MODEL, MOUSE, NORMAL, OF, Other, PROTEIN, PUBLISHED, RECIPIENTS, REPLACEMENT, Research, RESPONSES, RISK, secondary, STEM, STEM CELLS, STRATEGY, THE, THERAPY, TREATMENT, UK, Use, Animals Antigens/immunology Cells, Cultured Factor IX/*genetics/metabolism Gene Expression Gene Therapy/*methods Genetic Diseases, Inborn/genetics/therapy Genotype Green Fluorescent Proteins/genetics HIV-1/*genetics Hemophilia B/blood/*therapy Humans Immu
  • Addresses:
    1Gene Therapy Research Group, Faculty of Medicine, Imperial College London, South Kensington, London, UK
  • Notes:
    DA - 20060106IS - 0969-7128 (Print)LA - engPT - Journal ArticleSB - IM
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 IX, persisting for at least a year with tolerance to human factor IX antigen. Secondary and tertiary recipients also demonstrate long-term production of therapeutic levels of human factor IX and tolerance, even at very low levels of donor chimerism. Furthermore, in hemophilic mice, partial functional correction of treated mice and phenotypic rescue is achieved. These data show the potential of a stem cell approach to gene delivery to tolerize recipients to a secreted foreign transgenic protein and, with appropriate modification, may be of use in developing treatments for other genetic disorders.Gene Therapy (2006) 13, 117-126. doi:10.1038/sj.gt.3302638; published online 15 September 2005
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