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Publication Detail
Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.
  • Publication Type:
    Journal article
  • Publication Sub Type:
    Article
  • Authors:
    Waddington SN, Nivsarkar MS, Mistry AR, Buckley SM, Kemball-Cook G, Mosley KL, Mitrophanous K, Radcliffe P, Holder MV, Brittan M, Georgiadis A, Al-Allaf F, Bigger BW, Gregory LG, Cook HT, Ali RR, Thrasher A, Tuddenham EG, Themis M, Coutelle C
  • Publication date:
    01/11/2004
  • Pagination:
    2714, 2721
  • Journal:
    Blood
  • Volume:
    104
  • Issue:
    9
  • Print ISSN:
    0006-4971
  • Keywords:
    13, 14, ALL, AND, Antibodies, Antibody, ANTIGEN, ARTICLE, BLOOD, cell, CIRCULATION, development, DISEASE, enzyme, Enzymes, FACTOR IX, FETAL, GENE, Gene Therapy, GERMLINE, HEMOPHILIA, Hemophilia B, Human, HUMANS, Immunity, IS, JOURNAL, LA, LIVER, LONDON, MATERNAL, MEASUREMENT, MICE, MOLECULAR, Molecular Biology, MUTATION, MUTATIONS, NO, OF, PLASMA, Prenatal, PROPHYLAXIS, PROTEIN, Research, serum, SURGERY, THE, THERAPY, TREATMENT, UNITED-KINGDOM
Abstract
Hemophilia B, also known as Christmas disease, arises from mutations in the factor IX gene. Its treatment in humans, by recombinant protein substitution, is expensive thus limiting its application to intermittent treatment in bleeding episodes and prophylaxis during surgery; development of inhibitory antibodies is an associated hazard. This study demonstrates permanent therapeutic correction of this disease without development of immune reactions by introduction of an HIV-based lentiviral vector encoding the human factor IX protein into the fetal circulation of immunocompetent hemophiliac and normal outbred mice. Plasma factor IX antigen remained at around 9, 13 and 16% of normal in the three hemophilia B mice, respectively, until the last measurement at 14 months. Substantial improvement in blood coagulability as measured by coagulation assay was seen in all three mice and they rapidly stopped bleeding after venipuncture. No humoral or cellular immunity against the protein, elevation of serum liver enzymes or vector spread to the germline or maternal circulation were detected
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Institute of Ophthalmology
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Maternal & Fetal Medicine
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Infection, Immunity & Inflammation Dept
Author
Research Department of Haematology
Author
Maternal & Fetal Medicine
University College London - Gower Street - London - WC1E 6BT Tel:+44 (0)20 7679 2000

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