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Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.

Waddington, Simon N; Nivsarkar, Megha S; Mistry, Ajay R; Buckley, Suzanne M K; Kemball-Cook, Geoffrey; Mosley, Karen L; Mitrophanous, Kyriacos; Radcliffe, Pippa; Holder, Maxine V; Brittan, Mairi; Georgiadis, Anastasios; Al-Allaf, Faisal; Bigger, Brian W; Gregory, Lisa G; Cook, H Terence; Ali, Robin R; Thrasher, Adrian; Tuddenham, Edward G D; Themis, Mike; Coutelle, Charles

Blood. 2004;104(9):2714-21.

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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.

Bibliographic metadata

Content type:
Publication type:
Publication form:
Published date:
Language:
eng
Journal title:
Abbreviated journal title:
ISSN:
Place of publication:
United States
Volume:
104
Issue:
9
Pagination:
2714-21
Digital Object Identifier:
10.1182/blood-2004-02-0627
Pubmed Identifier:
15231566
Pii Identifier:
2004-02-0627
Access state:
Active

Institutional metadata

University researcher(s):

Record metadata

Manchester eScholar ID:
uk-ac-man-scw:56474
Created by:
Bigger, Brian
Created:
14th October, 2009, 10:17:01
Last modified by:
Bigger, Brian
Last modified:
4th May, 2010, 15:36:33

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