Adeno-associated virus-mediated gene transfer of factor IX for treatment of hemophilia B by gene therapy

Thrombosis and Haemostasis

Volumn 82, Issue 2, 1999, Pages 540-546

  Herzog, Roland W ^a   High, Katherine A ^a,b  

^a HOSPITAL OF THE UNIVERSITY OF PENNSYLVANIA   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL MODEL; CONFERENCE PAPER; GENE THERAPY; GENE TRANSFER; HEMOPHILIA B; HUMAN; NONHUMAN; PRIORITY JOURNAL;

EID: 0032748593     PISSN: 03406245     EISSN: None     Source Type: Journal    
DOI: 10.1055/s-0037-1615877     Document Type: Conference Paper

References (48)

1. Herzog RW, Yang EY, Couto LB, Hagstrom JN, Elwell D, Fields PA, Burton M, Bellinger DA, Read MS, Brinkhous KM, Podsakoff GM, Nichols TC, Kurtzman GJ, High KA. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat Med.. 1999;5:56-63.
2. Herzog RW, High KA. Problems and prospects in gene therapy for hemophilia. Curr Opin Hematol. 1998;5:321-326.
3. High KA. Gene therapy for hemophilia. New York: Wiley-Liss. 1998.
4. Evans JP, Brinkhous KM, Brayer GD, Reisner HM, High KA. Canine hemophilia B resulting from a point mutation with unusual consequences. Proc Natl Acad Sci USA. 1989;86:10095-10099.
5. Verma IM, Somia N. Gene therapy - promises, problems and prospects. Nature. 1997;389:239-242.
6. Kay MA, Meuse L, Gown AM, Linsley P, Hollenbaugh D, Aruffo A, Ochs HD, Wilson CB. Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver. Proc Natl Acad Sci USA.. 1997;94:4686-4691.
7. Fang B, Wang H, Gordon G, Bellinger DA, Read MS, Brinkhous KM, Woo SLC, Eisensmith RC. Lack of persistence of E1-recombinant adenoviral vectors containing a temperature-sensitive E2A mutation in immunocompetent mice and hemophilia B dog. Gene Ther. 1996;3:217-222.
8. Kay MA, Rothenberg S, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM, Woo SLC. In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs. Science. 1993;262:117-9.
9. Berns KI. Paroviridae: The viruses and their replication. 3rd ed. Philadelphia:Lippincott-Raven.1996.
10. Ferrari FK, Xiao X, McCarty D, Samulski RJ. New developments in the generation of Ad-free, high titer rAAV gene therapy vectors. Nat Med. 1997;3:1295-1297.
11. 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.
12. Matsushita T, Elliger S, Elliger C, Podsakoff G, Villarreal L, Kurtzman GJ, Iwaki Y, Colosi P. Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther. 1998;5:938-945.
13. Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol.. 1996;70:8098-8108.
14. Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Bryne BJ. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA.. 1996;93:14082-14087.
15. Fisher KJ, Jooss K, Alston J, Yang Y, Ehlen-Haecker S, High K, Pathak R, Raper SE, Wilson JM. Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med. 1997;3:306-312.
16. Clark KR, Sferra TJ, Johnson PR. Recombinant adeno-associated viral vectors mediate long-term transgene expression in muscle. Hum Gene Ther. 1997;8:659-669.
17. Snyder RO, Spratt SK, Lagarde C, Bohl D, Kaspar B, Sloan B, Cohen LK, Danos O. Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice. Hum Gene Ther. 1997b;8:1891-1900.
18. Jooss K, Yang Y, Fisher KJ, Wilson JM. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. J Virol.. 1998;72:4212-223.
19. Yao S-N, Smith KJ, Kurachi K. Primary myoblast-mediated gene transfer: persistent expression of human factor IX in mice. Gene Ther. 1994;1:99-107.
20. Podsakoff GM, Paszty C, Colosi PC, McQuiston SA, Kuypers FA, Witkowska HE, Mohandas N, Kurtzman GJ. Single dose, long-term treatment of beta-thalassemia in mice following intramuscular administration of the erythropoietin gene. Blood.. 1997;90(Suppl 1):1058.
21. Murphy JE, Zhou S, Giese K, Williams LT, Escobedo JA, Dwarki VJ. Long-term correction of obesity and diabetes in genetically obese mice by a single intramuscular injection of recombinant adeno-associated virus encoding mouse leptin. Proc Nat Acad Sci USA.. 1997;94:13921-13926.
22. Herzog RW, Hagstrom JN, Kung Z-H, Tai SJ, Wilson JM, Fisher KJ, High KA. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Natl Acad Sci USA.. 1997;94:5804-5809.
23. Fisher KJ, Gao G-P, Weitzman MD, DeMatteo R, Burda JF, Wilson JF. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol. 1996;70:520-532.
24. Koeberl DD, Alexander IE, Halbert CL, Russell DW, Miller AD. Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Natl Acad Sci USA. 1997;94:1426-1431.
25. Snyder RO, Miao CH, Patijn GA, Spratt SK, Danos O, Nagy D, Gown AM, Winther B, Meuse L, Cohen LK, Thompson AR, Kay MA. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet. 1997a;16:270-276.
26. Nakai H, Herzog R, Hagstrom JN, Kung J, Walter J, Tai S, Iwaki Y, Kurtzman G, Fisher K, Couto L, High KA. AAV-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood.. 1998;91:4600-4607.
27. Löser P, Jennings SG, Strauss M, Sandig V. Reactivation of the previously silenced cytomegalovirus major immediate-early promoter in the mouse liver: involvement of NFkB. J Virol. 1998;72:180-190.
28. Michou A, Santoro L, Christ M, Julliard V, Pavirani A, Mehtali M. Adenovirus-mediated gene transfer: influence of transgene, mouse strain and type of immune response on persistence of transgene expression. Gene Ther. 1997;4:473-482.
29. Miao CH, Snyder RO, Schowalter DB, Patijn GA, Donahue B, Winther B, Kay MA. The kinetics of rAAV integration in the liver. Nat Genet. 1998;19:13-15.
30. Duan D, Sharma P, Yang J, Yue Y, Dudus L, Fisher K, Engelhardt J. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long term episomal persistence in muscle. J Virol. 1998;72:8568-8577.
31. Kotin RM, Siniscalco M, Samulski RJ, Zhu D, Hunter L, Laughlin CA. Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA. 1990;87:2211-2215.
32. Weitzman MD, Kyostio SR, Kotin RM, Owens RA. Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA. Proc Natl Acad Scie USA. 1994;91:5808-58012.
33. Surosky RT, Urabe M, Godwin SG, McQuiston SA, Kurtzman GJ, Ozawa K, Natsoulis G. Adeno-associated virus rep proteins target DNA sequences to a unique locus in the human genome. J Virol. 1997;71:7951-7959.
34. Yang CG, Xiao X, Ansardi DC, Epstein ND, Frey MR, Matera AG, Samulski RJ. Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro. J Virol. 1997;71:9231-247.
35. Ponnazhagan S, Erikson D, Kearns WG, Zhou SZ, Nahreini P, Wang X-S, Srivastava A. Lack of site-specific integration of the recombinant adeno-associated virus 2 genomes in human cells. Hum Gene Ther. 1997;8:275-284.
36. Duan D, Fisher KJ, Burda JF, Engelhardt JF. Structural and functional heterogeneity of integrated recombinant AAV genomes. Virus Res. 1997;48:41-56.
37. Rutledge EA, Russell DW. Adeno-associated virus vector integration junctions. J Virol. 1997;71:8429-8436.
38. Cheung W-F, Born Jvd, Kühn K, Kjelléen L, Hudson BG, Stafford DW. Identification of the endothelial binding site for factor IX. Proc. Natl. Acad. Sci. USA.. 1996;93:11068-11073.
39. Lin H-F, Maeda N, Smithies O, Straight DL, Stafford DW. A coagulation factor IX-deficient mouse model for human hemophilia B. Blood. 1997;90:3962-3966.
40. Kung J, Hagstrom J, Cass D, Tai S, Lin HF, Stafford DW, High KA. Human FIX corrects the bleeding diathesis of mice with hemophilia B. Blood.. 1998;91:784-790.
41. Snyder RO, Miao C, Meuse L, Tubb J, Donahue BA, Lin H-F, Stafford DW, Patel S, Thompson AR, Nichols T, Read MS, Bellinger DA, Brinkhous KM, Kay MA. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med.1999;5:64-70.
42. High KA. Factor IX: molecular structure, epitopes, and mutations associated with inhibitor formation. In: Aledort LM, Hoyer LW, Lusher JM, Reisner HM, White GC, eds. Advances in Experimental Medicine and Biology. Vol. 386. New York: Plenum Press. 1995; 79-86.
43. Monahan PE, Samulski RJ, Tazelaar J, Xiao X, Nichols TC, Bellinger DA, Read MS. Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther.. 1998;5:40-49.
44. Tripathy SK, Black HB, Goldwasser E, Leiden JM. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nature Med.. 1996;2:545-550.
45. Bray GL, Gomperts ED, Courter S, Gruppo R, Gordon EM, Manco-Johnson M, Shapiro A, Scheibel E, White G, Lee M. A multicenter study of recombinant factor VIII (recombinate): Safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A. Blood. 1994;83:2428-2435.
46. Lusher JM, Arkin S, Abildgaard CF, Schwartz RS. Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. New Engl J Med. 1993;328:453-459.
47. Flotte T, Carter B, Conrad C, Guggino W, Reynolds T, Rosenstein B, Taylor G, Walden S, Wetzel R. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther. 1996;7:1145-1159.
48. Wagner JA, Moran ML, Messner AH, Daifuku R, Conrad CK, Reynolds T, Guggino WB, Moss RB, Carter BJ, Wine JJ, Flotte TR, Gardner P. A phase I/II study of tgAAV-CF for the treatment of chronic sinusitis in patients with cystic fibrosis. Hum Gene Ther. 1998;9:889-909.