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Blood ; 104(9): 2714-21, 2004 Nov 01.
Article in English | MEDLINE | ID: mdl-15231566

ABSTRACT

Hemophilia B, also known as Christmas disease, arises from mutations in the factor IX (F9) 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 his 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 3 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 3 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.


Subject(s)
Factor IX/administration & dosage , Fetal Therapies/methods , Genetic Therapy/methods , Hemophilia B/therapy , Animals , Blood Coagulation/drug effects , Factor IX/genetics , Factor IX/immunology , Female , Genetic Vectors/administration & dosage , Humans , Immune Tolerance , Immunocompetence , Lentivirus/genetics , Male , Mice , Mice, Inbred Strains , Phenotype , Placental Circulation , Pregnancy
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