Engineering of a stable retroviral gene delivery vector by directed evolution

Molecular Therapy

Volumn 16, Issue 2, 2008, Pages 308-314

  Vu, Halong N ^a   Ramsey, Joshua D ^a   Pack, Daniel W ^a  

^a UNIVERSITY OF ILLINOIS AT URBANA CHAMPAIGN   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PROTEINASE; RETROVIRUS VECTOR; VIRUS ENVELOPE PROTEIN;

AMINO ACID SUBSTITUTION; ARTICLE; CELL STRAIN 3T3; CELL STRAIN HEK293; CONTROLLED STUDY; DIRECTED MOLECULAR EVOLUTION; GENETIC ENGINEERING; GENETIC STABILITY; GENETIC TRANSDUCTION; HALF LIFE TIME; HUMAN; HUMAN CELL; INCUBATION TIME; MURINE LEUKEMIA VIRUS; MUTAGENESIS; NONHUMAN; VIRAL GENE DELIVERY SYSTEM; VIRUS GENOME; VIRUS INFECTIVITY; VIRUS MUTANT; VIRUS MUTATION; VIRUS RECOMBINANT; VIRUS REPLICATION; WILD TYPE;

MURINE LEUKEMIA VIRUS;

EID: 38649115377     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300350     Document Type: Article

References (34)

1. Somia, N and Verma, J (2000). Gene therapy: Trials and tribulations. Nat Genet 1: 91-99.
2. Thomas, CE, Ehrhardt, A and Kay, MA (2003). Progress and problems with the use of viral vectors for gene therapy. Nat Genet 4: 346-358.
3. Wickham, TJ (2003). Ligand-directed targeting of genes to the site of a disease. Nat Med 9: 135-139.
4. Cavazzana-Calvo, M, Hacein-Bey, S, de Saint Basile, G, Gross, F, Yvon, E, Nusbaum, P et al. (2000). Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669-672.
5. Aiuti, A, Slavin, S, Aker, M, Ficara, F, Deola, S, Mortellaro, A et al. (2002). Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296: 2410-2413.
6. Ott, MG, Schmidt, M, Schwarzwaelder, K, Stein, S, Siler, U, Koehl, U et al. (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 12: 401-409.
7. Andreadis, ST, Roth, CM, Le Doux, JM, Morgan, JR and Yarmush, ML (1999). Large-scale processing of recombinant retroviruses for gene therapy. Biotechnol Prog 15: 1-11.
8. Merten, OW (2004). State-of-the-art of the production of retroviral vectors. J Gene Med 6: S105-S124.
9. Powell, SK, Kaloss, MA, Pinkstaff, A, McKee, R, Burimski, I, Pensiero, M et al. (2000). Breeding of retroviruses by DNA shuffling for improved stability and processing yields. Nat Biotechnol 18: 1279-1282.
10. Anderson, WF (1998). Human gene therapy. Nature 392 (Suppl. 6679): 25-30.
11. Andreadis, ST, Brott, D, Fuller, AO and Palsson, BO (1997). Moloney murine leukemia virus-derived retroviral vectors decay intracellularly with a half-life in the range of 5.5 to 7.5 hours. J Virol 7l 7541-7548.
12. Le Doux, JM, Davis, HE, Morgan, JR and Yarmush, ML (1999). Kinetics of retrovirus production and decay. Biotechnol Bioeng 63: 654-662.
13. Coffin, JM (1996). Retroviridae: The viruses and their replication. In: Fields, BN, Knipe, DM and Howley, PM (eds). Fields Virology. Lippincott-Raven Publishers: Philadelphia, PA. pp. 1767-1847.
14. Pinter, A, Lieman-Hurvitz, J and Fleissner, E (1978). The nature of the association between the murine leukemia virus envelope proteins. Virology 91: 345-351.
15. Burns, JC, Friedmann, T, Driever, W, Burrascano, M and Yee, JK (1993). Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: Concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells. Proc Natl Acad Sci USA 90: 8033-8037.
16. Abe, A, Chen, ST, Miyanohara, A and Friedmann, T (1998). In vitro cell-free conversion of noninfectious Moloney retrovirus particles to an infectious form by the addition of the vesicular stomatitis virus surrogate envelope G protein. J Virol 72: 6356-6361.
17. Bassin, RH, Gerwin, BI, Levin, JG, Duran-Troise, G, Benjers, BM and Rein, A (1980). Macromolecular requirements for abrogation of Fv-1 restriction by murine leukemia viruses. J Virol 35: 287-297.
18. Arnold, FH (1998). Design by directed evolution. Acc Chem Res 31: 125-131.
19. Zhang, YX, Perry, K, Vinci, VA, Powell, K, Stemmer, WP and del Cardayre, SB (2002). Genome shuffling leads to rapid phenotypic improvement in bacteria. Nature 415: 644-646.
20. Low, NM, Holliger, P and Winter, G (1996). Mimicking somatic hypermutation: Affinity maturation of antibodies displayed on bacenophage using a bacterial mutator straih. J Mol Biol 260 359-368.
21. Kwon, YJ, Hung, G, Anderson, WF, Peng, CA and Yu, H (2003). Determination of infectious retrovirus concentration from colony-forming assay with quantitative analysis. J Virol 77: 5712-5720.
22. Menendez-Arias, L, Weber, IT and Oroszlan, S (1995). Mutational analysis of the substrate binding pocket of murine leukemia virus proease and comparison with human immunodeficiency virus proteases. J Biol Chem 270: 29162-29168.
23. Yoshinaka, Y, Katoh, I, Copeland, TD and Oroszlan, S (1985). Murine leukemia virus protease is encoded by the gag-pol gene and is synthesized through suppression of an amber termination codon. Proc Natl Acad Sci USA 82: 1618-1622.
24. Wlodawer, A and Gustchina, A (2000). Structural and biochemical studies of retroviral proteases. Biochim Biophys Acta 1477: 16-34.
25. Swanstrom, R and Wills, JW (1997). Synthesis, assembly, and processing of viral proteins. In: Coffin, JM, Hughes, SJ and Varmus, HE (eds). Retroviruses. Cold Spring Harbor: Plainview, NY pp. 263-334.
26. Weber, IT (1989). Structural alignment of retroviral protease sequences. Gene 85:565-566.
27. Pearl, LH and Taylor, LR (1987). A structural model for the retroviral proteases. Nature 329: 351-354.
28. Todd, MJ, Semo, N and Freire, E (1998). The structural stability of the HIV-1 protease. J Mol Biol 283: 475-488.
29. Kumar, M, Kannan, KK, Hosur, MV, Bhavesh, NS, Chatterjee, A, Mittal, R et al. (2002). Effects of remote mutation on the autolysis of HIV-1 PR: X-ray and NMR investigations. Biochim Biophys Acta 294: 395-401.
30. Layne, SP, Merges, MJ, Dembo, M, Spouge, JL, Conley, SR, Moore, JP et al. (1992). Factors underlying spontaneous inactivation and susceptibility to neutralizatoin of human immunodeficiency virus. Virology 189: 695-714.
31. Zhang, B, Metharom, P, Jullie, H, Ellem, KA, Cleghorn, G, West, MJ et al. (2004). The significance of controlled conditions in lentiviral vector titration and in the use of multiplicity of infection (MOI) for predicting gene transfer events. Genet Vaccines Ther 2; 6.
32. Flexner, C (1998). HIV-protease inhibitors. N Engl J Med 338 1281-1292.
33. Han, JY, Zhao, Y, Anderson, WF and Cannon, PM (1998). Role of variable regions A and B 'in receptor binding domain of amphotropic murine leukemia virus envelope protein. J Virol 72: 9101-9102.
34. Soneoka, Y, Cannon, PM, Ramsdale, EE, Griffiths, JC, Romano, G, Kingsman, SM et al. (1995). A transient three-plasmid expression system for the production of high titer retroviral vectors. Nucleic Acids Res 23: 628-633.