Design of modular non-viral gene therapy vectors

Biomaterials

Volumn 27, Issue 7, 2006, Pages 947-954

  De Laporte, Laura ^a   Cruz Rea, Jennifer ^a   Shea, Lonnie D ^a  

^a Northwestern University   (United States)

Author keywords

Cell trafficking; Gene therapy; Lipoplex; Polyplex; Vector

Indexed keywords

BIOMATERIALS; CATIONIC POLYMERIZATION; DRUG THERAPY; ENGINEERING RESEARCH; GENES; LIPIDS;

CELL TRAFFICKING; GENE THERAPY; LIPOPLEX; POLYPLEX;

VECTORS;

BETA CYCLODEXTRIN; BIOMATERIAL; CHITOSAN; CHLOROQUINE; DENDRIMER; DEXAMETHASONE; DIOLEOYLPHOSPHATIDYLETHANOLAMINE; ESTER DERIVATIVE; LIPOSOME; ORGANOPHOSPHATE; PEPTIDE NUCLEIC ACID; PLASMID DNA; POLY(2 DIETHYLAMINOETHYL METHACRYLATE); POLY(2 DIMETHYLAMINOETHYL METHACRYLATE); POLY(ETHYLENE OXIDE); POLY(PROPYLENE OXIDE); POLYAMIDOAMINE; POLYETHYLENEIMINE; POLYGLUCARAMIDOAMINE; POLYMER; POLYSACCHARIDE; PULLULAN; RECOMBINANT DNA; SPERMINE; UNCLASSIFIED DRUG; ZINC FINGER PROTEIN;

BIOTECHNOLOGY; DNA SEQUENCE; GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; GENE VECTOR; MEDICAL RESEARCH; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; PRIORITY JOURNAL; PROTEIN EXPRESSION; REVIEW; SUSTAINED DRUG RELEASE; TRANSGENE; ZETA POTENTIAL;

EID: 27644478375     PISSN: 01429612     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2005.09.036     Document Type: Article

References (54)

1. http://www.wiley.co.uk/wileychi/genmed/clinical/index.html. In.
2. J. Ziauddin, and D.M. Sabatini Microarrays of cells expressing defined cDNAs Nature 411 6833 2001 107 110
3. A. Martin-Herranz, A. Ahmad, H.M. Evans, K. Ewert, U. Schulze, and C.R. Safinya Surface functionalized cationic lipid-DNA complexes for gene delivery: PEGylated lamellar complexes exhibit distinct DNA-DNA interaction regimes Biophys J 86 2 2004 1160 1168
4. C. Boulanger, C. Di Giorgio, J. Gaucheron, and P. Vierling Transfection with fluorinated lipoplexes based on new fluorinated cationic lipids and in the presence of a bile salt surfactant Bioconjug Chem 15 4 2004 901 908
5. T. Segura, and L.D. Shea Materials for non-viral gene delivery Annu Rev Mater Res 31 2001 25 46
6. D.G. Anderson, D.M. Lynn, and R. Langer Semi-automated synthesis and screening of a large library of degradable cationic polymers for gene delivery Angew Chem Int Ed Engl 42 27 2003 3153 3158
7. K. Ewert, A. Ahmad, H.M. Evans, and C.R. Safinya Cationic lipid-DNA complexes for non-viral gene therapy: relating supramolecular structures to cellular pathways Expert Opin Biol Ther 5 1 2005 33 53
8. I. Tranchant, B. Thompson, C. Nicolazzi, N. Mignet, and D. Scherman Physicochemical optimisation of plasmid delivery by cationic lipids J Gene Med 6 Suppl 1 2004 S24 S35
9. O. Boussif, F. Lezoualc'h, M.A. Zanta, M.D. Mergny, D. Scherman, and B. Demeneix A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine Proc Natl Acad Sci USA 92 16 1995 7297 7301
10. C.S. Braun, J.A. Vetro, D.A. Tomalia, G.S. Koe, J.G. Koe, and C. Russell Middaugh Structure/function relationships of polyamidoamine/DNA dendrimers as gene delivery vehicles J Pharm Sci 94 2 2005 423 436
11. J. Wang, P.C. Zhang, H.Q. Mao, and K.W. Leong Enhanced gene expression in mouse muscle by sustained release of plasmid DNA using PPE-EA as a carrier Gene Ther 9 18 2002 1254 1261
12. A. Akinc, D.M. Lynn, D.G. Anderson, and R. Langer Parallel synthesis and biophysical characterization of a degradable polymer library for gene delivery J Am Chem Soc 125 18 2003 5316 5323
13. M. Neu, D. Fischer, and T. Kissel Recent advances in rational gene transfer vector design based on poly(ethylene imine) and its derivatives J Gene Med 7 8 2005 992 1009
14. D.V. Schaffer, N.A. Fidelman, N. Dan, and D.A. Lauffenburger Vector unpacking as a potential barrier for receptor-mediated polyplex gene delivery Biotechnol Bioeng 67 5 2000 598 606
15. A. Agarwal, R. Unfer, and S.K. Mallapragada Novel cationic pentablock copolymers as non-viral vectors for gene therapy J Control Release 103 1 2005 245 258
16. T.M. Reineke, and M.E. Davis Structural effects of carbohydrate- containing polycations on gene delivery. 1. Carbohydrate size and its distance from charge centers Bioconjug Chem 14 1 2003 247 254
17. S.R. Popielarski, S. Mishra, and M.E. Davis Structural effects of carbohydrate-containing polycations on gene delivery. 3. Cyclodextrin type and functionalization Bioconjug Chem 14 3 2003 672 678
18. Y. Liu, and T.M. Reineke Hydroxyl stereochemistry and amine number within poly(glycoamidoamine)s affect intracellular DNA delivery J Am Chem Soc 127 9 2005 3004 3015
19. K.W. Leong, H.Q. Mao, V.L. Truong-Le, K. Roy, S.M. Walsh, and J.T. August DNA-polycation nanospheres as non-viral gene delivery vehicles J Control Release 53 1-3 1998 183 193
20. M. Gupta, and A.K. Gupta Hydrogel pullulan nanoparticles encapsulating pBUDLacZ plasmid as an efficient gene delivery carrier J Control Release 99 1 2004 157 166
21. D.G. Anderson, A. Akinc, N. Hossain, and R. Langer Structure/property studies of polymeric gene delivery using a library of poly(beta-amino esters) Mol Ther 11 3 2005 426 434
22. S. Hwa Kim, J. Hoon Jeong, C.O. Joe, and T. Gwan Park Folate receptor mediated intracellular protein delivery using PLL-PEG-FOL conjugate J Control Release 103 3 2005 625 634
23. M. Ogris, S. Brunner, S. Schuller, R. Kircheis, and E. Wagner PEGylated DNA/transferring-PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery Gene Ther 6 4 1999 595 605
24. K. Kunath, T. Merdan, O. Hegener, H. Haberlein, and T. Kissel Integrin targeting using RGD-PEI conjugates for in vitro gene transfer J Gene Med 5 7 2003 588 599
25. T. Blessing, M. Kursa, R. Holzhauser, R. Kircheis, and E. Wagner Different strategies for formation of pegylated EGF-conjugated PEI/DNA complexes for targeted gene delivery Bioconjug Chem 12 4 2001 529 537
26. R. Kircheis, A. Kichler, G. Wallner, M. Kursa, M. Ogris, and T. Felzmann Coupling of cell-binding ligands to polyethylenimine for targeted gene delivery Gene Ther 4 5 1997 409 418
27. A. Kichler Gene transfer with modified polyethylenimines J Gene Med 6 Suppl 1 2004 S3 S10
28. A.K. Pannier, and L.D. Shea Controlled release systems for DNA delivery Mol Ther 10 1 2004 19 26
29. H.J. Kong, J. Liu, K. Riddle, T. Matsumoto, K. Leach, and D.J. Mooney Non-viral gene delivery regulated by stiffness of cell adhesion substrates Nat Mater 2005
30. J.H. Jang, C.B. Rives, and L.D. Shea Plasmid delivery in vivo from porous tissue engineering scaffolds: transgene expression and cellular transfection Mol Ther 12 3 2005 475 483
31. Y.C. Huang, K. Riddle, K.G. Rice, and D.J. Mooney Long-term in vivo gene expression via delivery of PEI-DNA condensates from porous polymer scaffolds Hum Gene Ther 16 5 2005 609 617
32. F. Scherer, U. Schillinger, U. Putz, A. Stemberger, and C. Plank Nonviral vector loaded collagen sponges for sustained gene delivery in vitro and in vivo J Gene Med 4 6 2002 634 643
33. J. Zhang, L.S. Chua, and D.M. Lynn Multilayered thin films that sustain the release of functional DNA under physiological conditions Langmuir 20 19 2004 8015 8021
34. B. Bajaj, P. Lei, and S.T. Andreadis High efficiencies of gene transfer with immobilized recombinant retrovirus: kinetics and optimization Biotechnol Prog 17 4 2001 587 596
35. A.K. Pannier, B.C. Anderson, and L.D. Shea Substrate-mediated delivery from self-assembled monolayers: effect of surface ionization, hydrophilicity, and patterning Acta Biomater 1 5 2005 511 522
36. T. Segura, M.J. Volk, and L.D. Shea Substrate-mediated DNA delivery: role of the cationic polymer structure and extent of modification J Control Release 93 1 2003 69 84
37. T. Segura, P.H. Chung, and L.D. Shea DNA delivery from hyaluronic acid-collagen hydrogels via a substrate-mediated approach Biomaterials 26 13 2005 1575 1584
38. C.M. Varga, N.C. Tedford, M. Thomas, A.M. Klibanov, L.G. Griffith, and D.A. Lauffenburger Quantitative comparison of polyethylenimine formulations and adenoviral vectors in terms of intracellular gene delivery processes Gene Ther 2005
39. M.C. Morris, L. Chaloin, F. Heitz, and G. Divita Translocating peptides and proteins and their use for gene delivery Curr Opin Biotechnol 11 5 2000 461 466
40. C.M. Roth, and S. Sundaram Engineering synthetic vectors for improved DNA delivery: insights from intracellular pathways Annu Rev Biomed Eng 6 2004 397 426
41. A. Kichler, K. Mechtler, J.P. Behr, and E. Wagner Influence of membrane-active peptides on lipospermine/DNA complex mediated gene transfer Bioconjug Chem 8 2 1997 213 221
42. T.B. Wyman, F. Nicol, O. Zelphati, P.V. Scaria, C. Plank, and F.C. Szoka Jr. Design, synthesis, and characterization of a cationic peptide that binds to nucleic acids and permeabilizes bilayers Biochemistry 36 10 1997 3008 3017
43. M. Baru, O. Nahum, H. Jaaro, J. Sha'anani, and I. Nur Lysosome-disrupting peptide increases the efficiency of in-vivo gene transfer by liposome-encapsulated DNA J Drug Target 6 3 1998 191 199
44. D.A. Dean, D.D. Strong, and W.E. Zimmer Nuclear entry of nonviral vectors Gene Ther 12 11 2005 881 890
45. Y. Chen, P. Lenert, R. Weeratna, M. McCluskie, T. Wu, and H.L. Davis Identification of methylated CpG motifs as inhibitors of the immune stimulatory CpG motifs Gene Ther 8 13 2001 1024 1032
46. S. Li, S.P. Wu, M. Whitmore, E.J. Loeffert, L. Wang, and S.C. Watkins Effect of immune response on gene transfer to the lung via systemic administration of cationic lipidic vectors Am J Physiol 276 5 Pt 1 1999 L796 L804
47. J.A. Gruneich, A. Price, J. Zhu, and S.L. Diamond Cationic corticosteroid for nonviral gene delivery Gene Ther 11 8 2004 668 674
48. X. Zhang, G.J. Sawyer, X. Dong, Y. Qiu, L. Collins, and J.W. Fabre The in vivo use of chloroquine to promote non-viral gene delivery to the liver via the portal vein and bile duct J Gene Med 5 3 2003 209 218
49. S.A. Kelkar, K.K. Pfister, R.G. Crystal, and P.L. Leopold Cytoplasmic dynein mediates adenovirus binding to microtubules J Virol 78 18 2004 10122 10132
50. E. Check Harmful potential of viral vectors fuels doubts over gene therapy Nature 423 6940 2003 573 574
51. F.D. Urnov, J.C. Miller, Y.L. Lee, C.M. Beausejour, J.M. Rock, and S. Augustus Highly efficient endogenous human gene correction using designed zinc-finger nucleases Nature 2005
52. Y. Liu, L. Wenning, M. Lynch, and T.M. Reineke New poly(d- glucaramidoamine)s induce DNA nanoparticle formation and efficient gene delivery into mammalian cells J Am Chem Soc 126 24 2004 7422 7423
53. W. Ritter, C. Plank, J. Lausier, C. Rudolph, D. Zink, and D. Reinhardt A novel transfecting peptide comprising a tetrameric nuclear localization sequence J Mol Med 81 11 2003 708 717
54. K.H. Bremner, L.W. Seymour, A. Logan, and M.L. Read Factors influencing the ability of nuclear localization sequence peptides to enhance nonviral gene delivery Bioconjug Chem 15 1 2004 152 161