Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

Blood

Volumn 107, Issue 7, 2006, Pages 2653-2661

  Nathwani, Amit C ^a   Gray, John T ^b   Ng, Catherine Y C ^b   Zhou, Junfang ^b   Spence, Yunyu ^b   Waddington, Simon N ^b   Tuddenham, Edward G D ^b   Kemball Cook, Geoffrey ^b   McIntosh, Jenny ^b   Boon Spijker, Mariette ^b   Mertens, Koen ^b   Davidoff, Andrew M ^b  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9;

ANIMAL EXPERIMENT; ARTICLE; BLEEDING TENDENCY; GENE THERAPY; GENETIC TRANSDUCTION; HEMOPHILIA B; LIVER; MALE; MOUSE; NONHUMAN; PARVOVIRUS VECTOR; PRIMATE; PRIORITY JOURNAL;

EID: 33645528206     PISSN: 00064971     EISSN: 00064971     Source Type: Journal    
DOI: 10.1182/blood-2005-10-4035     Document Type: Article

References (38)

1. Nathwani AC, Tuddenham EG. Epidemiology of coagulation disorders. Baillieres Clin Haematol. 1992;5:383-439.
2. Nathwani AC, Davidoff AM, Tuddenham EG. Prospects for gene therapy of haemophilia. Haemophilia. 2004;10:309-318.
3. Fisher KJ, Gao GP, Weitzman MD, et al. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol. 1996;70:520-532.
4. Nakai H, Storm TA, Kay MA. Annealing of complimentary single stranded genomes and subsequent intermolecular joining is the mechanism of stable in vivo liver transduction by recombinant adeno-associated virus vectors [abstract]. Mol Ther. 2000;1:S125-S126.
5. Weitzman MD, Fisher KJ, Wilson JM. Recruitment of wild-type and recombinant adeno-associated virus into adenovirus replication centers. J Virol. 1996;70:1845-1854.
6. Alexander IE, Russell DW, Miller AD. DNA-damaging agents greatly increase the transduction of non-dividing cells by adeno-associated virus vectors. J Virol. 1994;68:8282-8287.
7. Mah C, Qing K, Khuntirat B, et al. Adeno-associated virus type 2-mediated gene transfer: role of epidermal growth factor receptor protein tyrosine kinase in transgene expression. J Virol. 1998;72:9835-9843.
8. Gao GP, Alvira MR, Wang L, et al. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A. 2002;99:11854-11859.
9. Thomas CE, Storm TA, Huang Z, Kay MA. Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol. 2004;78:3110-3122.
10. Davidoff AM, Gray JT, Ng CY, et al. Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5 and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models. Mol Ther. 2005;11:875-888.
11. Nakai H, Fuess S, Storm TA, et al. Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol. 2005;79:214-224.
12. High K, Tigges M, Manno CS, et al. Human immune responses to AAV-2 capsid may limit duration of expression in liver-directed gene transfer in humans with hemophilia B [abstract]. Blood. 2004;104:121a.
13. Nathwani AC, Davidoff A, Hanawa H, et al. Factors influencing in-vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. Blood. 2001;97:1258-1265.
14. McCarty DM, Fu H, Monahan PE, et al. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther. 2003;10:2112-2118.
15. Wang Z, Ma HI, Li J, et al. Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 2003;10:2105-2111.
16. Hirata RK, Russell DW. Design and packaging of adeno-associated virus gene targeting vectors. J Virol. 2000;74:4612-4620.
17. Yang GS, Schmidt M, Yan Z, et al. Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size. J Virol. 2002;76:7651-7660.
18. Haas J, Park EC, Seed B. Codon usage limitation in the expression of HIV-1 envelope glycoprotein. Curr Biol. 1996;6:315-324.
19. Davidoff AM, Ng CY, Sleep S, et al. Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock. J Virol Methods. 2004;121:209-215.
20. Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol. 1998;72:2224-2232.
21. Chiorini JA, Kim F, Yang L, Kotin RM. Cloning and characterization of adeno-associated virus type 5. J Virol. 1999;73:1309-1319.
22. Rabinowitz JE, Rolling F, Li C, et al. Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J Virol. 2002;76:791-801.
23. Wang L, Zoppe M, Hackeng TM, et al. A factor IX-deficient mouse model for hemophilia B gene therapy. Proc Natl Acad Sci U S A. 1997;94:11563-11566.
24. Nakai H, Yant SR, Storm TA, et al. Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol. 2001;75:6969-6976.
25. Nathwani AC, Davidoff AM, Hanawa H, et al. Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques. Blood. 2002;100:1662-1669.
26. Waddington SN, Nivsarkar MS, Mistry AR, et al. Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. Blood. 2004;104:2714-2721.
27. Davidoff AM, Ng CY, Zhou J, Spence Y, Nathwani AC. Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood. 2003;102:480-488.
28. Nathwani AC, Hanawa H, Vandergriff J, et al. Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV. Gene Ther. 2000;7:183-195.
29. Christophe OD, Lenting PJ, Cherel G, et al. Functional mapping of anti-factor IX inhibitors developed in patients with severe hemophilia B. Blood. 2001;98:1416-1423.
30. Rohlena J, Kolkman JA, Boertjes RC, Mertens K, Lenting PJ. Residues Phe342-Asn346 of activated coagulation factor IX contribute to the interaction with low density lipoprotein receptor-related protein. J Biol Chem. 2003;278:9394-9401.
31. Lozier JN, Metzger ME, Donahue RE, Morgan RA. The rhesus macaque as an animal model for hemophilia B gene therapy. Blood. 1999;93:1875-1881.
32. Miao CH, Ohashi K, Patijn GA, et al. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Mol Ther. 2000;1:522-532.
33. Green PM, Saad S, Lewis CM, Giannelli F. Mutation rates in humans, I: overall and sex-specific rates obtained from a population study of hemophilia B. Am J Hum Genet. 1999;65:1572-1579.
34. Samulski RJ, Srivastava A, Berns KI, Muzyczka N. Rescue of adeno-associated virus from recombinant plasmids: gene correction within the terminal repeats of AAV. Cell. 1983;33:135-143.
35. High K, Manno CS, Sabatino DE, et al. Immune response to AAV and to factor IX in a phase I study of AAV-mediated liver directed gene transfer for hemophilia B [abstract]. Mol Ther. 2004;9: S383-S384.
36. Nakai H, Thomas CE, Storm TA, et al. A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction. J Virol. 2002;76:11343-11349.
37. High KA, Manno CS, Sabatino DE, et al. Immune responses to AAV and to factor IX in a phase I study of AAV-mediated liver-directed gene transfer for hemophilia B [abstract]. Blood. 2003;102:154a-155a.
38. Stasi R, Brunetti M, Stipa E, Amadori S. Selective B-cell depletion with rituximab for the treatment of patients with acquired hemophilia. Blood. 2004;103:4424-4428.