Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: Implications for gene therapy

Human Gene Therapy

Volumn 10, Issue 14, 1999, Pages 2295-2305

  Loeb, Jonathan E ^a,b   Cordier, Wendy S ^a   Harris, Matthew E ^a   Weitzman, Matthew D ^a   Hope, Thomas J ^a  

^a SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; GREEN FLUORESCENT PROTEIN; VIRUS VECTOR;

ADENOVIRUS; ARTICLE; FIBROBLAST CULTURE; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSDUCTION; HEPATITIS VIRUS; HUMAN; HUMAN CELL; NONHUMAN; REPORTER GENE; TRANSCRIPTION REGULATION; TRANSGENE; VIRUS RECOMBINANT;

BLOTTING, WESTERN; CELL LINE; DEPENDOVIRUS; FLOW CYTOMETRY; GENE EXPRESSION REGULATION, VIRAL; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEPATITIS B VIRUS, WOODCHUCK; HUMANS; LUMINESCENT PROTEINS; REGULATORY SEQUENCES, NUCLEIC ACID; RNA PROCESSING, POST-TRANSCRIPTIONAL; RNA, VIRAL; TRANSGENES;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; DNA VIRUSES; MARMOTA MONAX; WOODCHUCK HEPATITIS B VIRUS;

EID: 0033588890     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950016942     Document Type: Article

References (35)

1. ANTONIOU, M., GERAGHTY, F., HURST, J., and GROSVELD, F. (1998). Efficient 3′-end formation of human beta-globin mRNA in vivo requires sequences within the last intron but occurs independently of the splicing reaction. Nucleic Acids Res. 26, 721-729.
2. BUCHMAN, A.R., and BERG, P. (1988). Comparison of intron-dependent and intron-independent gene expression. Mol. Cell. Biol. 8, 4395-4405.
3. CARTER, B.J. (1992). Adeno-associated virus vectors. Curr. Opin. Biotechnol. 3, 533-539.
4. CHEUNG, A.K., HOGGAN, M.D., HAUSWIRTH, W.W., and BERNS, K.I. (1980). Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells. J. Virol. 33, 739-748.
5. CHOI, T., HUANG, M., GORMAN, C., and JAENISCH, R. (1991). A generic intron increases gene expression in transgenic mice. Mol. Cell. Biol. 11, 3070-3074.
6. DONELLO, J.E., BEECHE, A.A., SMITH, G.R., LUCERO, G.R., and HOPE, T.J., (1996). The hepatitis B virus posttranscriptional regulatory element is composed of two subelements. J. Virol. 70, 4345-4351.
7. DONELLO, J.E., LOEB, J.E., and HOPE, T.J. (1998). Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. J. Virol. 72, 5085-5092.
8. DONG, J.Y., FAN, P.D., and FRIZZELL, R.A. (1996). Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum. Gene Ther. 7, 2101-2112.
9. FISCHER, U., MEYER, S., TEUFEL, M., HECKEL, C., LUHRMANN, R., and RAUTMANN, G. (1994). Evidence that HIV-1 Rev directly promotes the nuclear export of unspliced RNA. EMBO J. 13, 4105-4112.
10. FISHER, K.J., GAO, G.P., WEITZMAN, M.D., DEMATTEO, R., BURDA, J.F., and WILSON, J.M. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70, 520-532.
11. FLOTTE, T.R., and CARTER, B.J. (1995). Adeno-associated virus vectors for gene therapy. Gene Ther. 2, 357-362.
12. FLOTTE, T.R., AFIONE, S.A., CONRAD, C., MCGRATH, S.A., SOLOW, R., OKA, H., ZEITLIN, P.L., GUGGINO, W.B., and CARTER, B.J. (1993). Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. U.S.A. 90, 10613-10617.
13. HARRIS, M.E., DONELLO, J.E., LOEB, J.E., OTERO, G.C., and HOPE, T.J. (1999). A cis-acting RNA element that increases expression by promoting longer polyadenylated tails. (In preparation.)
14. HUANG, Y., WIMLER, K.M., and CARMICHAEL, G.G. (1999). Intronless mRNA transport elements may affect multiple steps of pre-mRNA processing. EMBO J. 18, 1642-1652.
15. HUANG, Z.M., and YEN, T.S. (1994). Hepatitis B virus RNA element that facilitates accumulation of surface gene transcripts in the cytoplasm. J. Virol. 68, 3193-3199.
16. HUANG, Z.M., and YEN, T.S. (1995). Role of the hepatitis B virus posttranscriptional regulatory element in export of intronless transcripts. Mol. Cell. Biol. 15, 3864-3869.
17. KOTIN, R.M. (1994). Prospects for the use of adeno-associated virus as a vector for human gene therapy. Hum. Gene Ther. 5, 793-801.
18. LAUGHLIN, C.A., CARDELLICHIO, C.B., and COON, H.C. (1986). Latent infection of KB cells with adeno-associated virus type 2. J. Virol. 60, 515-524.
19. MALIM, M.H., HAUBER, J., LE, S.Y., MAIZEL, J.V., and CULLEN, B.R. (1989). The HIV-1 Rev trans-activator acts through a structured target sequence to activate nuclear export of unspliced viral mRNA. Nature (London) 338, 254-257.
20. MCLAUGHLIN, S.K., COLLIS, P., HERMONAT, P.L., and MUZYCZKA, N. (1988). Adeno-associated virus general transduction vectors: Analysis of proviral structures. J. Virol. 62, 1963-1973.
21. MURPHY, J.E., ZHOU, S., GIESE, K., WILLIAMS, L.T., ESCOBEDO, J.A. and DWARKI, V.J. (1997). 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. Natl. Acad. Sci. U.S.A. 94, 13921-13926.
22. NESIC, D., CHENG, J., and MAQUAT, L.E. (1993). Sequences within the last intron function in RNA 3′-end formation in cultured cells. Mol. Cell. Biol. 13, 3359-3369.
23. PASQUINELLI, A.E., ERNST, R.K., LUND, E., GRIMM, C., ZAPP, M.L., REKOSH, D., HAMMARSKJOLD, M.L., and DAHLBERG, J.E. (1997). The constitutive transport element (CTE) of Mason-Pfizer monkey virus (MPMV) accesses a cellular mRNA export pathway. EMBO J. 16, 7500-7510.
24. RYU, W.S., and MERTZ, J.E. (1989). Simian virus 40 late transcripts lacking excisable intervening sequences are defective in both stability in the nucleus and transport to the cytoplasm. J. Virol. 63, 4386-4394.
25. SAAVEDRA, C., FELBER, B., and IZAURRALDE, E. (1997). The simian retrovirus-1 constitutive transport element, unlike the HIV-1 RRE, uses factors required for cellular mRNA export. Curr. Biol. 7, 619-628.
26. SAMULSKI, R.J., CHANG, L.S., and SHENK, T. (1987). 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. 61, 3096-3101.
27. SAMULSKI, R.J., CHANG, L.S., and SHENK, T. (1989). Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression. J. Virol. 63, 3822-3828.
28. SMITH, G.J., III, DONELLO, J.E., LÜCK, R., STEGER, G., and HOPE, T.J. (1998). The hepatitis B virus post-transcriptional regulatory element contains two conserved RNA stem-loops which are required for function. Nucleic Acids Res. 26, 4818-4827.
29. SNYDER, R.O., MIAO, C.H., PATIJN, G.A., SPRATT, S.K., DANOS, O., NAGY, D., GOWN, A.M., WINTHER, B., MEUSE, L., COHEN, L.K., THOMPSON, A.R., and KAY, M.A. (1997). Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genet. 16, 270-276.
30. TSIEN, R. (1998). The green fluorescent protein. Annu. Rev. Biochem. 67, 509-544.
31. WANG, J.M., ZHENG, H., SUGAHARA, Y., TAN, J., YAO, S.N., OLSON, E., and KURACHI, K. (1996). Construction of human factor IX expression vectors in retroviral vector frames optimized for muscle cells. Hum. Gene Ther. 7, 1743-1756.
32. XIAO, X., LI, J., and SAMULSKI, R.J. (1996). Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70, 8098-8108.
33. XIAO, X., LI, J., MCCOWN, T.J., and SAMULSKI, R.J. (1997). Gene transfer by adeno-associated virus vectors into the central nervous system. Exp. Neurol. 144, 113-24.
34. XIAO, X., LI, J., and SAMULSKI, R.J. (1998). Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72, 2224-2232.
35. ZUFFEREY, R., DONELLO, J.E., TRONO, D., and HOPE, T.J. (1999). Woodchuck hepatitis virus post-transcriptional regulatory element (WPRE) enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73, 2886-2892.