Adeno-associated viral vectors: Background and technical aspects

Experimental Nephrology

Volumn 5, Issue 6, 1997, Pages 514-520

  Snoeck, Hans Willem ^a   Tao, Wenjing ^a   Klotman, Mary E ^a  

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus; Gene therapy; Vector production

Indexed keywords

VIRUS DNA; VIRUS VECTOR;

ADENOVIRUS; CONFERENCE PAPER; GENE THERAPY; NONHUMAN; PARVOVIRUS; PRIORITY JOURNAL; VIRUS CONCENTRATION; VIRUS RECOMBINANT;

DEPENDOVIRUS; DNA, RECOMBINANT; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS;

EID: 0031456167     PISSN: 10187782     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Conference Paper

References (44)

1. Miller AD: Human gene therapy comes of age. Nature 1992;357:455-460.
2. Mulligan R: The basic science of gene therapy. Science 1993;260:926-932.
3. Condon C, Watkins SC, Celluzzi CM, Thompson K, Falo LD Jr: DNA-based immunization by in vivo transfection of dendritic cells. Nat Med 1996;2:1122-1128.
4. Miller GD, Adam MA, Miller AD: Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Moll Cell Biol 1990;10:4239-4242.
5. Dunckley MG, Wells DJ, Walsh FS, Dickson S: Direct retroviral-mediated transfer of a dystrophin minigene into mdx mouse muscle in vivo. Hum Moll Genet 1993;2:717-723.
6. Ogawa M: Differentiation and proliferation of hematopoietic stem cells. Blood 1993;81:2844.
7. Wang Q, Finer MI: Second generation adenovirus vectors. Nat Med 1996;2:714-716.
8. Berns KI, Rose JA: Evidence for a single stranded adenovirus-associated virus genome: Isolation and separation of complementary single strands. J Virol 1970;5:693-699.
9. Kotin RM, Siniscalco M, Samulski RJ, Zhu X, Hunter L, Laughlin CA, McLaughlin S, Muzycka N, Rocchi L, Berns KI: Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 1990;87:2211-2215.
10. Kotin RM, Linden RM, Berns KI: Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. EMBO J 1992;11:5071-5078.
11. Samulski RJ, Zhu X, Xiao X, Brook JD, Housman DE, Epstein N, Hunter LA: Targeted integration of adeno-associated virus (AAV) into chromosome 19. EMBO J 1991;10:3941-3950.
12. Buller RM, Janik JA, Sebring ED, Rose JA: Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication. J Virol 1981;40:241-247.
13. Janik JE, Huston MM, Rose JA: Locations of adenovirus genes required for replication of adeno-associated virus. Proc Natl Acad Sci USA 1981;78:1925-1930.
14. Muzyczka N: In vitro replication of adeno-associated virus DNA. Semin Virol 1991;2: 281-290.
15. Kotin RM: Prospects for the use of adeno-associated virus as a vector for human gene therapy. Hum Gene Ther 1994;5:793-801.
16. Shaugnessy E, Lu D, Chatterjee S, Wong KK: Parvoviral vectors for gene therapy of cancer. Semin Oncol 1996;23:159-171.
17. Xiao X, Xiao W, Li J, Samulski RJ: A novel 165-base-pair terminal repeat sequence is the sole cis requirement for the adeno-associated virus life cycle. J Virol 1997;71:941-948.
18. Ferrari FK, Samulski T, Shenk T, Samulski RJ: Second-strand synthesis is a rate limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 1996;70: 3227-3234.
19. Fisher KJ, Gao G, Weitzman MD, DeMatteo R, Burda JF, Wilson JM: Transduction with recombinant adeno-associated virus vectors for gene therapy is limited by leading strand synthesis. J Virol 1996;70:520-532.
20. Samulski RJ, Chang L, Shenk T: A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J Virol 1987;61:3096-3101.
21. Nahreini P, Woody WJ, Zhou SZ, Srivastava A: Versatile adeno-associated virus-2 vectors for constructing recombinant virions. Gene 1993;124:257-262.
22. Samulski RJ, Chang L, Shenk T: Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression. J Virol 1989;63:3822-3828.
23. Ficher KJ, Jooss K, Alston J, Yang Y, Haecker SE, High K, Pathak R, Raper SE, Wilson JM: Recombinant adeno-associatcd virus for muscle directed gene therapy. Nat Med 1997;3: 306-312.
24. 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.
25. Walsh CE, Nienhuis AW, Samulski RJ, Brown MG, Miller JL, Young NS, Liu JM: Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J Clin Invest 1994;94: 1440-1448.
26. Malik P, McQuiston SA, Yu X, Pepper KA, Krall WJ, Podsakoff GM, Kurtzman GJ, Kohn DB: Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line. J Virol 1997;71:1776-1783.
27. Fisher-Adams G, Wong KK Jr, Podsakoff G, Forman SJ, Chatterjee S: Integration of adeno-associatcd virus vectors in CD34+ human hematopoietic progenitor cells after transduction. Blood 1996;88:492-504.
28. Flotte TR, Afione SA, Conrad C, McGrath SA, Solow R, Oka H, Zeitlin PL, Guggino WB, Carter BJ: Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci USA 1993;90:10613-10617.
29. Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, O Malley KL, During MJ: Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 1994;8:148-153.
30. Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byrne BJ: Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 1996;93:14982-14987.
31. Russel DW, Alexander IE, Miller AD: DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors. Proc Natl Acad Sci USA 1995;92: 5719-5723.
32. Rivadeneira ED, Popescu NC, Zimonjic D, Ross MD, Cheng GS, DiPaolo JA, Gallo RC, Klotman ME: Recombinant adeno-associated virus for gene delivery: Transduction and integration. AIDS Res Hum Retroviruses 1994; 10(suppl 3):159.
33. Kearns WG, Afione SA, Fulmer SB, Pang MG, Erikson D, Egan M, Landrum M, Flotte TR, Cutting GR: Recombinant adeno-associated virus (AAV-CFTR) does not integrate in a site-specific fashion in an immortalized epithelial cell line. Gene Ther 1996;3:748-755.
34. Ponnazhagan S, Erikson D, Kearns WG, Zhou SZ, Nahreini P, Wang X, Srivatava A: Lack of site-specific integration of the recombinant adeno-associated genomes in human cells. Hum Gene Ther 1997;8:275-284.
35. Fan P, Dong J: Replication of rep-cap genes is essential for the high-efficiency production of recombinant AAV. Hum Gene Ther 1997;8: 87-98.
36. Hermonat PL: Down-regulation of the human c-fos and c-myc proto-oncogene promoters by adeno-associated virus rep 78. Cancer Lett 1994;81:129-136.
37. Hölscher C, Hörer M, Kleinschmidt JA, Zentgraf H, Bürkle A, Heilbronn R: Cell lines inducibly expressing the adeno-associated virus (AAV) rep-gene: Requirements for productive replication of rep-negative AAV-mutants. J Virol 1994;68:7169-7177.
38. Yang Q, Chen F, Trempe JP: Characterization of cell lines that inducibly express the adeno-associated virus rep-proteins. J Virol 1994;68: 4847-4856.
39. Tamayose K, Hirai Y, Shimada T: A new strategy for large scale preparation of high-titer recombinant adeno-associated virus vectors by using packaging cell lines and sulfonated cellulose column chromatography. Hum Gene Ther 1996;7:507-513.
40. Vincent KA, Piraino ST, Wadsworth SC: Analysis of recombinant adeno-associated virus packaging and requirements for rep and cap gene products. J Virol 1997;71:1897-1905.
41. Podsakoff G, Wong KK Jr, Chatterjee S: Efficient gene transfer into nondividing cells by adeno-associated virus-based vectors. J Virol 1994;68:5656-5666.
42. Flotte TR, Afione SA, Zeitlin PL: Adeno-associated virus vector gene expression occurs in non-dividing cells in the absence of vector integration. Am J Respir Cell Mol Biol 1994;11: 517-521.
43. Russel DW, Miller AD, Alexander IE: Adeno-associated virus vectors preferentially transduce cells in S-phase. Proc Natl Acad Sci USA 1994;91:8915-8919.
44. Alexander IE, Russel DW, Miller AD: DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors. J Virol 1994;68:8282-8287.