Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV)

Journal of Gene Medicine

Volumn 2, Issue 6, 2000, Pages 444-454

  O'Riordan, Catherine R ^a   Lachapelle, Amy L ^a   Vincent, Karen A ^a   Wadsworth, Samuel C ^a  

^a GENZYME CORPORATION   (United States)

Author keywords

Adeno associated virus; Gene therapy; Vector purification

Indexed keywords

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; GOSSYPIUM HIRSUTUM; PARVOVIRUS; SIGMODON;

BETA GALACTOSIDASE; HYBRID PROTEIN; RECOMBINANT DNA;

ADENO ASSOCIATED VIRUS; ANIMAL; ARTICLE; BAGG ALBINO MOUSE; CELL LINE; CHROMATOGRAPHY; GENE EXPRESSION REGULATION; GENE TRANSFER; GENE VECTOR; GENETICS; HUMAN; ION EXCHANGE CHROMATOGRAPHY; ISOLATION AND PURIFICATION; LUNG; METABOLISM; METHODOLOGY; MOUSE; SIGMODONTINAE; VIROLOGY;

ANIMALS; BETA-GALACTOSIDASE; CELL LINE; CHROMATOGRAPHY; CHROMATOGRAPHY, ION EXCHANGE; DEPENDOVIRUS; DNA, RECOMBINANT; GENE EXPRESSION REGULATION; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; LUNG; MICE; MICE, INBRED BALB C; RECOMBINANT FUSION PROTEINS; SIGMODONTINAE;

EID: 0034328476     PISSN: 1099498X     EISSN: None     Source Type: Journal    
DOI: 10.1002/1521-2254(200011/12)2:6<444::AID-JGM132>3.0.CO;2-1     Document Type: Article

References (39)

1. Flotte TR, Afione SA, Conrad C, et al. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci U S A 1993; 90: 10613-10617.
2. Kaplitt MG, Leone P, Samulski RJ, et al. Long-term gene expression and phenotypic correction using an adeno-associated virus vector in the mammalian brain. Nat Genet 1994; 8: 148-154.
3. Kessler PD, Podsakoff GM, Chen X, et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci U S A 1996; 93: 14082-14087.
4. Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompotent mice by adeno-associated virus vector. J Virol 1996; 70: 8098-8108.
5. Clark KR, Sferra TJ, Johnson PR. Recombinant adeno-associated virus vectors mediate long-term transgene expression in muscle. Hum Gen Ther 1997; 8: 659-669.
6. Fisher KJ, Jooss K, Alston J, et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med 1997; 3: 306-312.
7. Flannery JG, Zolotukhin S, Vaquero MI, et al. Efficient photoreceptor targeted gene expression in vivo by recombinant adeno-associated virus. Proc Natl Acad Sci U S A 1997; 94: 6916-6921.
8. Koeberl DD, Alexander IE, Halbert CL, et al. Persistent expression of human clotting facter IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Natl Acad Sci U S A 1997; 94: 1426-1431.
9. Snyder RO, Miao CH, Patjin GA, et al. Persistent and therapeutic concentration of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet 1997; 16: 270-276.
10. Greelish JP, Su LT, Lankford EB, et al. Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector. Nat Med 1999; 5: 439-443.
11. Dudus L, Anand V, Acland GM, et al. Persistent transgene product in retina, optic nerve and brain after intraocular injection of rAAV. Vision Res 1999; 39: 2545-2553.
12. Bennett J, Maguire AM, Cideciyan AV, et al. Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natl Acad Sci U S A 1999; 96: 9920-9925.
13. McCown TJ, Xiao X, Li J, et al. Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res 1996; 713: 99-107.
14. Flotte T, Carter B, Conrad C, et al. 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.
15. Miao CH, Snyder RO, Schowalter DB, et al. The kinetics of rAAV integration in the liver. Nat Genet 1998; 19: 13-15.
16. 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: 4212-4223.
17. Samulski RJ, Chang LS, Shenk T. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol 1989; 61: 3096-3101.
18. Flotte TR, Barraza-Ortiz X, Solow R, et al. An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction. Gene Ther 1995; 1: 29-37.
19. Li J, Samulski RJ, Xiao X. Role of highly regulated rep gene expression in adeno-associated virus vector production. J Virol 1997; 71: 5236-5243.
20. 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.
21. Grimm DK, Kern A, Rittner K, Kleinschimdt JA. Novel tools for production and purification of recombinant AAV vectors. Hum Gene Ther 1998; 9: 2745-2760.
22. 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.
23. Gao GP, Qu G, Faust LZ, et al. High-titer adeno-associated viral vectors from a Rep/Cap cell line and hybrid shuttle virus. Hum Gen Ther 1998; 9: 2353-2362.
24. Bartlett JS, Xiao X, Samulski RJ. Adeno-associated virus vectors for gene transfer. In Protocols for Gene Transfer in Neuroscience: Towards Gene Therapy of Neurological Disorders, Loewestein PR, Enquist LW (eds). Wiley: New York, 1996; 115-130.
25. Snyder RO, Xiao X, Samulski RJ. Production of recombinant adeno-associated viral vectors. In Current Protocols in Human Genetics, Dracopoli N (ed.). Wiley: New York, 1996; 12.1.1-12.1.20.
26. Hermens W, Brake OT, Duzhuizen PA, et al. Purification of recombinant adeno-associated virus by iodixanol gradient ultracentrifugation allows rapid and reproducible preparation of vector stocks for gene transfer in the nervous system. Hum Gene Ther 1999; 10: 1885-1891.
27. Zolotukhin S, Byrne BJ, Mason E, et al. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther 1999; 6: 973-985.
28. Clark RK, Liu X, McGrath JP, Johnson PR. Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses. Hum Gene Ther 1999; 10: 1031-1039.
29. Graham FL, Smiley J, Russel WC, Nairn R. Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J Gen Virol 1977; 36: 59-74.
30. Einsinger MJ, Ginsberg, HS. Selection and preliminary characterization of temperature-sensitive mutants of type 5 adenovirus. J Virol 1972; 10: 328-339.
31. Stillman BW, Tamanoi F, Mathews MB. Purification of an adenovirus-coded DNA polymerase that is required for initiation of DNA replication. Cell 1982; 31: 613-623.
32. Chejanovsky N, Carter BJ. Replication of a human parvovirus nonsense mutant in mammalian cells containing an inducible amber suppressor. Virology 1989; 171: 239-247.
33. Lynn DE. A BASIC computer program for analyzing endpoint assays. BioTechniques 1992; 12: 880-881.
34. Rich DP, Couture LA, Cardoza LM, et al. Development and analysis of recombinant adenoviruses for gene therapy of cystic fibrosis. Hum Gene Ther 1993; 4: 461-476.
35. Huyghe BG, Liu X, Sutjipto S, et al. Purification of a type 5 recombinant adenovirus encoding human p53 by column chromatography. Hum Gene Ther 1995; 11: 1403-1416.
36. Cotmore SF, Tattersall P. The autonomously replicating parvoviruses of vertebrates. Adv Virus Res 1987; 33: 91-97.
37. Summerford C, Samulski RJ. Membrane-associated heparan sulphate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 1998; 72: 1438-1445.
38. 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.
39. 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.