NLS bioconjugates for targeting therapeutic genes to the nucleus

Advanced Drug Delivery Reviews

Volumn 55, Issue 2, 2003, Pages 295-306

  Escriou, Virginie ^a   Carrière, Marie ^a   Scherman, Daniel ^a   Wils, Pierre ^b  

^a SANOFI   (France)

^b Gencell SAS   (France)

Author keywords

Gene transfer; Non viral; Nuclear import; Plasmid DNA

Indexed keywords

DNA; GENES; PROTEINS; RNA;

CYTOPLASM;

DRUG PRODUCTS;

NUCLEAR PROTEIN; NUCLEIC ACID; PLASMID DNA;

CELL NUCLEUS; CONJUGATE; GENE DELIVERY SYSTEM; GENE TARGETING; PRIORITY JOURNAL; PROTEIN ANALYSIS; REVIEW; SEQUENCE ANALYSIS; VIRUS;

EID: 0347926089     PISSN: 0169409X     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0169-409X(02)00184-9     Document Type: Article

References (43)

1. Kay M.A., Glorioso J.C., Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat. Med. 7:2001;33-40.
2. Nishikawa M., Huang L. Non-viral vectors in the new millennium: delivery barriers in gene transfer. Hum. Gene Ther. 12:2001;861-870.
3. Zabner J., Fasbender A.J., Moninger T., Poellinger K.A., Welsh M.J. Cellular and molecular barriers to gene transfer by a cationic lipid. J. Biol. Chem. 270:1995;18997-19007.
4. Pedroso de Lima M.C., Simoes S., Pires P., Faneca H., Duzgunes N. Cationic lipid-DNA complexes in gene delivery: from biophysics to biological applications. Adv. Drug Deliv. Rev. 47:2001;277-294.
5. Hwang S.J., Davis M.E. Cationic polymers for gene delivery: designs for overcoming barriers to systemic administration. Curr. Opin. Mol. Ther. 3:2001;183-191.
6. Capecchi M.R. High efficiency transformation by direct microinjection of DNA into cultured mammalian cells. Cell. 22:1980;479-488.
7. Escriou V., Carrière M., Bussone F., Wils P., Scherman D. Critical assessment of the nuclear import of plasmid during cationic lipid-mediated gene transfer. J. Gene Med. 3:2001;179-187.
8. D. Lechardeur, K.J. Sohn, M. Haardt, P.B. Joshi, M. Monck, R.W. Graham, B. Beatty, J. Squire, H. O'Brodovich, G.L. Lukacs, Metabolic instability of plasmid DNA in the cytosol: a potential barrier to gene transfer, Gene Ther. 6 (1999).
9. Lukacs G.L., Haggie P., Seksek O., Lechardeur D., Freedman N., Verkman A. Size-dependent DNA mobility in cytoplasm and nucleus. J. Biol. Chem. 275:2000;1625-1629.
10. Hagstrom J.E., Ludtke J.J., Bassik M.C., Sebestyén M.G., Adam S.A., Wolff J.A. Nuclear import of DNA in digitonin-permeabilized cells. J. Cell Sci. 110:1997;2323-2331.
11. Dean D.A. Import of plasmid DNA into the nucleus is sequence specific. Exp. Cell Res. 230:1997;293-302.
12. Wilson G.L., Dean B.S., Wang G., Dean D.A. Nuclear import of plasmid DNA in digitonin-permeabilized cells requires both cytoplasmic factors and specific DNA sequences. J. Biol. Chem. 274:1999;22025-22032.
13. Längle-Rouault F., Patzel V., Benavente A., Taillez M., Silvestre N., Bompard A., Sczakiel G., Jacobs E., Rittner K. Up to 100-fold increase of apparent gene expression in the presence of Epstein-Barr virus oriP sequences and EBNA1: implications of the nuclear import of plasmids. J. Virol. 72:1998;6181-6185.
14. Vacik J., Dean B.S., Zimmer W.E., Dean D.A. Cell-specific nuclear import of plasmid DNA. Gene Ther. 6:1999;1006-1014.
15. Conti E., Izaurralde E. Nucleocytoplasmic transport enters the atomic age. Curr. Opin. Cell Biol. 13:2001;310-319.
16. Ryan K.J., Wente S.R. The nuclear pore complex: a protein machine bridging the nucleus and cytoplasm. Curr. Opin. Cell Biol. 12:2000;361-371.
17. Feldherr C.M., Akin D. The permeability of the nuclear envelope in dividing and non-dividing cells. J. Cell Biol. 111:1998;1-8.
18. Imamoto N. Diversity in nucleocytoplasmic transport pathways. Cell Struct. Funct. 25:2000;207-216.
19. Whittaker G.R., Kann M., Helenius A. Viral entry into the nucleus. Annu. Rev. Cell Dev. Biol. 16:2000;627-651.
20. Goff S.P. Intracellular trafficking of retroviral genomes during the early phase of infection: viral exploitation of cellular pathways. J. Gene Med. 3:2001;517-528.
21. Whittaker G.R., Helenius A. Nuclear import and export of viruses and virus genomes. Virology. 246:1998;1-23.
22. O'Neill R.E., Jaskunas R., Blobel G., Palese P., Moroianu J. Nuclear import of influenza virus RNA can be mediated by viral nucleoprotein and transport factors required for protein import. J. Biol. Chem. 270:1995;22701-22704.
23. Trotman L.C., Mosberger N., Fornerod M., Stidwill R.P., Greber U.F. Import of adenovirus DNA involves the nuclear pore complex receptor CAN/Nup214 and histone H1. Nat. Cell Biol. 3:2001;1092-1100.
24. Wienhues U., Hosokawa K., Höveler A., Siegmann B., Doerfler W. A novel method for transfection and expression of reconstituted DNA-protein complexes in eukaryotic cells. DNA. 6:1987;81-89.
25. Kaneda Y., Iwai K., Uchida T. Increased expression of DNA cointroduced with nuclear protein in adult rat liver. Science. 243:1989;375-378.
26. Ziemienowicz A., Görlich D., Lanka E., Hohn B., Rossi L. Import of DNA into mammalian nuclei by proteins originating from a plant pathogenic bacterium. Proc. Natl. Acad. Sci. USA. 96:1999;3729-3733.
27. Landford R.E., Butel J.S. Construction and characterization of an SV40 mutant defective in nuclear transport of T antigen. Cell. 37:1984;801-813.
28. Collas P., Husebye H., Aleström P. The nuclear localization sequence of the SV40 T antigen promotes transgene uptake and expression in zebrafish embryo nuclei. Transgenic Res. 5:1996;451-458.
29. Collas P., Aleström P. Nuclear localization signal of SV40 T antigen directs import of plasmid DNA into sea urchin male pronuclei in vitro. Mol. Reprod. Dev. 45:1996;431-438.
30. Aronsohn A.I., Hughes J.A. Nuclear localization signal peptides enhance cationic liposome-mediated gene therapy. J. Drug Target. 5:1997;163-169.
31. Ogris M., Steinlein P., Kursa M., Mechtler K., Kircheis R., Wagner E. The size of DNA/transferrin-PEI complexes is an important factor for gene expression in cultured cells. Gene Ther. 5:1998;1425-1433.
32. Escriou V., Ciolina C., Lacroix F., Byk G., Scherman D., Wils P. Cationic-lipid mediated gene transfer: effect of serum on cellular uptake and intracellular fate of lipopolyamine/DNA complexes. Biochim. Biophys. Acta. 1368:1998;276-288.
33. Schwartz B., Ivanov M.-A., Pitard B., Escriou V., Rangara R., Byk G., Wils P., Crouzet J., Scherman D. Synthetic DNA-compacting peptides derived from human sequence enhance cationic lipid-mediated gene transfer in vitro and in vivo. Gene Ther. 6:1999;282-292.
34. Subramanian A., Ranganathan P., Diamond S.L. Nuclear targeting peptide scaffolds for lipofection of non-dividing mammalian cells. Nat. Biotechnol. 17:1999;873-877.
35. Brandén L.J., Mohamed A.J., Smith C.I.E. A peptide nucleic acid-nuclear localization signal fusion that mediates nuclear transport of DNA. Nat. Biotechnol. 17:1999;784-787.
36. Ludtke J.J., Zhang G., Sebestyén M.G., Wolff J.A. A nuclear localization signal can enhance both the nuclear transport and expression of 1 kb DNA. J. Cell Sci. 112:1999;2033-2041.
37. Sebestyén M.G., Ludtke J.J., Bassik M.C., Zhang G., Budker V., Lukhtanov E.A., Hagstrom J.E., Wolff J.A. DNA vector chemistry: the covalent attachment of signal peptides to plasmid DNA. Nat. Biotechnol. 16:1998;80-85.
38. Zanta M.-A., Belguise-Valladier P., Behr J.-P. Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc. Natl. Acad. Sci. USA. 96:1999;91-96.
39. Ciolina C., Byk G., Blanche F., Thuillier V., Scherman D., Wils P. Coupling of nuclear localization signals to plasmid DNA and specific interaction of the conjugates with importin alpha. Bioconjug. Chem. 10:1999;49-55.
40. Neves C., Byk G., Scherman D., Wils P. Coupling of a targeting peptide to plasmid DNA by covalent triple helix formation. FEBS Lett. 453:1999;41-45.
41. Rebuffat A., Bernasconi A., Ceppi M., Wehrli H., Brenz Verca S., Ibrahim M., Frey B.M., Rusconi S. Selective enhancement of gene transfer by steroid-mediated gene delivery. Nat. Biotechnol. 19:2001;1155-1161.
42. Cutrona G., Carpaneto E.M., Ulivi M., Roncella S., Landt O., Ferrarini M., Boffa L.C. Effects in live cells of a c-myc anti-gene PNA linked to a nuclear localization signal. Nat. Biotechnol. 18:2000;300-303.
43. Brandén L.J., Christensson B., Smith C.I.E. In vivo nuclear delivery of oligonucleotides via hybridizing bifunctional peptides. Gene Ther. 8:2001;84-87.