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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
biochemistry, genetics and molecular biology
Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy
Blood, Volume 104, No. 9, Year 2004
Notification
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Description
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 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 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. © 2004 by The American Society of Hematology.
Authors & Co-Authors
Waddington, Simon N.
United Kingdom, London
Imperial College London
Nivsarkar, Megha S.
Unknown Affiliation
Buckley, Suzanne M.K.
Unknown Affiliation
Holder, Maxine V.
Unknown Affiliation
Al-Allaf, Faisal Ahmad
Unknown Affiliation
Bigger, Brian W.
Unknown Affiliation
Gregory, Lisa G.
Unknown Affiliation
Ali, Robin R.
Unknown Affiliation
Thrasher, Adrian James
Unknown Affiliation
Tuddenham, Edward G.D.
Unknown Affiliation
Themis, Michael
Unknown Affiliation
Coutelle, Charles C.
Unknown Affiliation
Statistics
Citations: 116
Authors: 12
Affiliations: 1
Identifiers
Doi:
10.1182/blood-2004-02-0627
ISSN:
00064971
Research Areas
Environmental
Genetics And Genomics
Health System And Policy
Infectious Diseases
Maternal And Child Health