Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector

Nature Genetics

Volumn 24, Issue 3, 2000, Pages 257-261

  Kay, Mark A ^a   Manno, Catherine S ^b,c   Ragni, Margaret V ^d   Larson, Peter J ^b,c   Couto, Linda B ^e   McClelland, Alan ^e   Glader, Bertil ^a   Chew, Amy J ^b   Tai, Shing Jen ^b   Herzog, Roland W ^b   Arruda, Valder ^b   Johnson, Fred ^e   Scallan, Ciaran ^e   Skarsgard, Erik ^a   Flake, Alan W ^b,c   High, Katherine A ^b,c  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

^d UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^e Avigen Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9 COMPLEX; VIRUS VECTOR;

ADENOVIRUS; ADULT; AGED; ANTIBODY TITER; ARTICLE; BLOOD CLOTTING; CASE REPORT; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DOSE RESPONSE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HEMOPHILIA B; HUMAN; HUMAN TISSUE; IMMUNE RESPONSE; PHASE 1 CLINICAL TRIAL; PRIORITY JOURNAL;

ADULT; AGED; BLOOD COAGULATION TESTS; BLOTTING, SOUTHERN; DEPENDOVIRUS; FACTOR IX; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA B; HUMANS; INJECTIONS, INTRAMUSCULAR; MALE; MUSCLE, SKELETAL; POLYMERASE CHAIN REACTION; RECOMBINANT FUSION PROTEINS; TREATMENT OUTCOME;

ADENOVIRIDAE; ANIMALIA; CANIS FAMILIARIS;

EID: 0034050904     PISSN: 10614036     EISSN: None     Source Type: Journal    
DOI: 10.1038/73464     Document Type: Article

References (16)

1. Herzog, R.W. et al. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl Acad. Sci. USA 94, 5804-5809 (1997).
2. Herzog, R. et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nature Med. 5, 56-63 (1999).
3. Ljung, R.C.R. Annotation. Can haemophilic arthropathy be prevented? Br. J. Haematol. 101, 215-219 (1998).
4. Lofqvist, T., Nilsson, I.M., Berntorp, E. & Pettersson, H. Haemophilia prophylaxis in young patients - a long term follow up. J. Intern. Med. 241, 395-400 (1997).
5. Mannucci, P.M. & Weitz, J.I. The hemophilias: progress and problems. Semin. Hematol. 36, 104-117 (1999).
6. Parks, W.P., Boucher, D.W., Melnick, J.L., Taber, L.H. & Yow, M.D Seroepidemiological and ecological studies of the adenovirus-associated satellite viruses. Infect. Immun. 2, 716-722 (1970).
7. Summerford, C. & Samulski, R.J. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol. 72, 1438-1445 (1998).
8. Qing, K. et al. Human fibroblast growth growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nature Med. 5, 71-77 (1999).
9. Cheung, W.F. et al. Identification of the endothelial cell binding site for factor IX. Proc. Natl Acad. Sci. USA 93, 11068-11073 (1996).
10. Matsushita, T. et al. Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther. 5, 938-945 (1998).
11. Burton, M. et al. Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein. Proc. Natl Acad. Sci. USA 96, 12725-12730 (1999).
12. Yoshitake, S., Schach, B.G., Foster, D.C., Davie, E.W. & Kuracki, K. Nucleotide sequence of the gene for human factor IX (antihemophilic factor B). Biochemistry 24, 3736-3750 (1985).
13. Kurachi, S., Hitomi, Y., Midori, F. & Kurachi, K. Role of intron I in expression of the human factor IX gene. J. Biol. Chem. 270, 5276-5281 (1995).
14. Manno, C.S., Larson, P.J., Cohen, A.R. & Flake, A.W. Guidelines for Research Involving Recombinant DNA Molecules (NIH Guidelines). Appendix M. Points to consider in the design and submission of protocols for the transfer of recombinant DNA molecules into one or more human subjects. (NIH Recombinant DNA Advisory Committee, Washington DC, 1999).
15. Walter, J., You, Q., Hagstrom, J.N., Sands, M. & High, K.A. Successful expression of human factor IX following repeat administration of adenoviral vector in mice. Proc. Natl Acad. Sci. USA 93, 3056-3061 (1996).
16. Miller, J.H. Experiments in Molecular Genetics 352-355 (Cold Spring Harbor Press, Cold Spring Harbor, New York, 1972).