Peptide-mediated DNA condensation for non-viral gene therapy

Biotechnology Advances

Volumn 27, Issue 4, 2009, Pages 432-438

  Saccardo, Paolo ^a   Villaverde, Antonio ^a,b   González Montalbán, Nuria ^a,b  

^a UNIVERSITAT AUTÒNOMA DE BARCELONA   (Spain)

^b BIOMATERIALES Y NANOMEDICINA CIBER BBN   (Spain)

Author keywords

Artificial viruses; Cationic peptides; DNA binding; Gene therapy; Multifunctional proteins; Nanoparticles; Protein engineering; Recombinant drugs

Indexed keywords

ARTIFICIAL VIRUSES; CATIONIC PEPTIDES; DNA BINDING; GENE THERAPY; MULTIFUNCTIONAL PROTEINS; PROTEIN ENGINEERING; RECOMBINANT DRUGS;

AMINES; BIOCHEMICAL ENGINEERING; DNA; DYES; GENE TRANSFER; GENES; MICROORGANISMS; NANOPARTICLES; NUCLEIC ACIDS; PEPTIDES;

DRUG THERAPY;

ARGININE; CATION; DNA; DNA BINDING PROTEIN; NANOPARTICLE; PEPTIDE; POLYLYSINE;

BIOACTIVITY; CATION; CONDENSATION; DNA; DRUG DEVELOPMENT; GENE EXPRESSION; GENETIC ENGINEERING; PEPTIDE; RECOMBINATION; VIRUS;

CHEMISTRY; GENE THERAPY; GENE TRANSFER; METABOLISM; METHODOLOGY; PROTEIN BINDING; REVIEW;

ARGININE; CATIONS; DNA; DNA-BINDING PROTEINS; GENE THERAPY; GENE TRANSFER TECHNIQUES; NANOPARTICLES; PEPTIDES; POLYLYSINE; PROTEIN BINDING;

EID: 67349279624     PISSN: 07349750     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.biotechadv.2009.03.004     Document Type: Review

References (63)

1. Abbott A. Questions linger about unexplained gene-therapy trial death. Nat Med 12 (2006) 597
2. Aris A., and Villaverde A. Molecular organization of protein-DNA complexes for cell-targeted DNA delivery. Biochem Biophys Res Commun 278 (2000) 455-461
3. Aris A., and Villaverde A. Engineering nuclear localization signals in modular protein vehicles for gene therapy. Biochem Biophys Res Commun 304 (2003) 625-631
4. Aris A., and Villaverde A. Modular protein engineering for non-viral gene therapy. Trends Biotechnol 22 (2004) 371-377
5. Aris A., Feliu J.X., Knight A., Coutelle C., and Villaverde A. Exploiting viral cell-targeting abilities in a single polypeptide, non-infectious, recombinant vehicle for integrin-mediated DNA delivery and gene expression. Biotechnol Bioeng 68 (2000) 689-696
6. Astriab-Fisher A., Sergueev D., Fisher M., Shaw B.R., and Juliano R.L. Conjugates of antisense oligonucleotides with the Tat and antennapedia cell-penetrating peptides: effects on cellular uptake, binding to target sequences, and biologic actions. Pharm Res 19 (2002) 744-754
7. Bloomfield V.A. DNA condensation. Curr Opin Struct Biol 6 (1996) 334-341
8. Bloomfield V.A. DNA condensation by multivalent cations. Biopolymers 44 (1997) 269-282
9. Brewer L.R., Corzett M., and Balhorn R. Protamine-induced condensation and decondensation of the same DNA molecule. Science 286 (1999) 120-123
10. Capito R.M., and Spector M. Collagen scaffolds for nonviral IGF-1 gene delivery in articular cartilage tissue engineering. Gene Ther 14 (2007) 721-732
11. Chan C.K., Hubner S., Hu W., and Jans D.A. Mutual exclusivity of DNA binding and nuclear localization signal recognition by the yeast transcription factor GAL4: implications for nonviral DNA delivery. Gene Ther 5 (1998) 1204-1212
12. Choi Y.H., Liu F., Kim J.S., Choi Y.K., Park J.S., and Kim S.W. Polyethylene glycol-grafted poly-l-lysine as polymeric gene carrier. J Control Release 54 (1998) 39-48
13. Cohen-Sacks H., Elazar V., Gao J., Golomb A., Adwan H., Korchov N., et al. Delivery and expression of pDNA embedded in collagen matrices. J Control Release 95 (2004) 309-320
14. Dai H., Choe W.S., Thai C.K., Sarikaya M., Traxler B.A., Baneyx F., et al. Nonequilibrium synthesis and assembly of hybrid inorganic-protein nanostructures using an engineered DNA binding protein. J Am Chem Soc 127 (2005) 15637-15643
15. Edelstein M.L., Abedi M.R., and Wixon J. Gene therapy clinical trials worldwide to 2007-an update. J Gene Med 9 (2007) 833-842
16. Facchini L.M., and Lingwood C.A. A verotoxin 1 B subunit-lambda CRO chimeric protein specifically binds both DNA and globotriaosylceramide (Gb(3)) to effect nuclear targeting of exogenous DNA in Gb(3) positive cells. Exp Cell Res 269 (2001) 117-129
17. Ferrer-Miralles N., Vazquez E., and Villaverde A. Membrane-active peptides for non-viral gene therapy: making the safest easier. Trends Biotechnol 26 (2008) 267-275
18. Gottschalk S., Sparrow J.T., Hauer J., Mims M.P., Leland F.E., Woo S.L., et al. A novel DNA-peptide complex for efficient gene transfer and expression in mammalian cells. Gene Ther 3 (1996) 448-457
19. Haines A.M., Irvine A.S., Mountain A., Charlesworth J., Farrow N.A., Husain R.D., et al. CL22 - a novel cationic peptide for efficient transfection of mammalian cells. Gene Ther 8 (2001) 99-110
20. He S., Arscott P.G., and Bloomfield V.A. Condensation of DNA by multivalent cations: experimental studies of condensation kinetics. Biopolymers 53 (2000) 329-341
21. Henke E., Perk J., Vider J., de C.P., Chin Y., Solit D.B., et al. Peptide--conjugated antisense oligonucleotides for targeted inhibition of a transcriptional regulator in vivo. Nat Biotechnol 26 (2008) 91-100
22. Ignatovich I.A., Dizhe E.B., Pavlotskaya A.V., Akifiev B.N., Burov S.V., Orlov S.V., et al. Complexes of plasmid DNA with basic domain 47-57 of the HIV-1 Tat protein are transferred to mammalian cells by endocytosis-mediated pathways. J Biol Chem 278 (2003) 42625-42636
23. Jin X., Mei L., Song C., Liu L., Leng X., Sun H., et al. Immobilization of plasmid DNA on an anti-DNA antibody modified coronary stent for intravascular site-specific gene therapy. J Gene Med 10 (2008) 421-429
24. Keller M., Harbottle R.P., Perouzel E., Colin M., Shah I., Rahim A., et al. Nuclear localisation sequence templated nonviral gene delivery vectors: investigation of intracellular trafficking events of LMD and LD vector systems. Chembiochem 4 (2003) 286-298
25. Kim H.H., Lee W.S., Yang J.M., and Shin S. Basic peptide system for efficient delivery of foreign genes. Biochim Biophys Acta 1640 (2003) 129-136
26. Kim H.H., Choi H.S., Yang J.M., and Shin S. Characterization of gene delivery in vitro and in vivo by the arginine peptide system. Int J Pharm 335 (2007) 70-78
27. Kumar P., Wu H., McBride J.L., Jung K.E., Kim M.H., Davidson B.L., et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature 448 (2007) 39-43
28. Lamaziere A., Wolf C., Lambert O., Chassaing G., Trugnan G., and yala-Sanmartin J. The homeodomain derived peptide Penetratin induces curvature of fluid membrane domains. PLoS ONE 3 (2008) e1938
29. Lechardeur D., Sohn K.J., Haardt M., Joshi P.B., Monck M., Graham R.W., et al. Metabolic instability of plasmid DNA in the cytosol: a potential barrier to gene transfer. Gene Ther 6 (1999) 482-497
30. Lee C.J., Paik S.H., Ko K.H., and Kim K.C. Effects of polycationic peptides on mucin release from airway goblet cells: relationship between polymer size and activity. Inflamm Res 51 (2002) 490-494
31. Lee H., Jeong J.H., and Park T.G. PEG grafted polylysine with fusogenic peptide for gene delivery: high transfection efficiency with low cytotoxicity. J Control Release 79 (2002) 283-291
32. Li Z., and Huang L. Sustained delivery and expression of plasmid DNA based on biodegradable polyester, poly(d,l-lactide-co-4-hydroxy-l-proline). J Control Release 98 (2004) 437-446
33. Manickam D., Bisht H., Wan L., Mao G., and Oupicky D. Influence of TAT-peptide polymerization on properties and transfection activity of TAT/DNA polyplexes. J Control Release 102 (2005) 293-306
34. Mannisto M., Vanderkerken S., Toncheva V., Elomaa M., Ruponen M., Schacht E., et al. Structure-activity relationships of poly(l-lysines): effects of pegylation and molecular shape on physicochemical and biological properties in gene delivery. J Control Release 83 (2002) 169-182
35. Marshall E. Gene therapy. What to do when clear success comes with an unclear risk?. Science 298 (2002) 510-511
36. Marshall E. Gene therapy. Second child in French trial is found to have leukemia. Science 299 (2003) 320
37. Martin M.E., and Rice K.G. Peptide-guided gene delivery. AAPS J 9 (2007) E18-E29
38. Mastrobattista E., van der Aa M.A., Hennink W.E., and Crommelin D.J. Artificial viruses: a nanotechnological approach to gene delivery. Nat Rev Drug Discov 5 (2006) 115-121
39. McKenzie D.L., Collard W.T., and Rice K.G. Comparative gene transfer efficiency of low molecular weight polylysine DNA-condensing peptides. J Pept Res 54 (1999) 311-318
40. McKenzie D.L., Kwok K.Y., and Rice K.G. A potent new class of reductively activated peptide gene delivery agents. J Biol Chem 275 (2000) 9970-9977
41. McKenzie D.L., Smiley E., Kwok K.Y., and Rice K.G. Low molecular weight disulfide cross-linking peptides as nonviral gene delivery carriers. Bioconjug Chem 11 (2000) 901-909
42. Mirny L.A., and Gelfand M.S. Structural analysis of conserved base pairs in protein-DNA complexes. Nucleic Acids Res 30 (2002) 1704-1711
43. Moschos S.A., Jones S.W., Perry M.M., Williams A.E., Erjefalt J.S., Turner J.J., et al. Lung delivery studies using siRNA conjugated to TAT(48-60) and penetratin reveal peptide induced reduction in gene expression and induction of innate immunity. Bioconjug Chem 18 (2007) 1450-1459
44. Peluffo H., Aris A., Acarin L., Gonzalez B., Villaverde A., and Castellano B. Nonviral gene delivery to the central nervous system based on a novel integrin-targeting multifunctional protein. Hum Gene Ther 14 (2003) 1215-1223
45. Peluffo H., Acarin L., Aris A., Gonzalez P., Villaverde A., Castellano B., et al. Neuroprotection from NMDA excitotoxic lesion by Cu/Zn superoxide dismutase gene delivery to the postnatal rat brain by a modular protein vector. BMC Neurosci 7 (2006) 35
46. Plank C., Tang M.X., Wolfe A.R., and Szoka Jr. F.C. Branched cationic peptides for gene delivery: role of type and number of cationic residues in formation and in vitro activity of DNA polyplexes. Hum Gene Ther 10 (1999) 319-332
47. Putnam D., Gentry C.A., Pack D.W., and Langer R. Polymer-based gene delivery with low cytotoxicity by a unique balance of side-chain termini. Proc Natl Acad Sci U S A 98 (2001) 1200-1205
48. Read M.L., Bremner K.H., Oupicky D., Green N.K., Searle P.F., and Seymour L.W. Vectors based on reducible polycations facilitate intracellular release of nucleic acids. J Gene Med 5 (2003) 232-245
49. Rimann M., Luhmann T., Textor M., Guerino B., Ogier J., and Hall H. Characterization of PLL-g-PEG-DNA nanoparticles for the delivery of therapeutic DNA. Bioconjug Chem 19 (2008) 548-557
50. Symonds P., Murray J.C., Hunter A.C., Debska G., Szewczyk A., and Moghimi S.M. Low and high molecular weight poly(l-lysine)s/poly(l-lysine)-DNA complexes initiate mitochondrial-mediated apoptosis differently. FEBS Lett 579 (2005) 6191-6198
51. Tecle M., Preuss M., and Miller A.D. Kinetic study of DNA condensation by cationic peptides used in nonviral gene therapy: analogy of DNA condensation to protein folding. Biochemistry 42 (2003) 10343-10347
52. Tolmachov O., and Coutelle C. Covalent attachment of multifunctional chimeric terminal proteins to 5′ DNA ends: a potential new strategy for assembly of synthetic therapeutic gene vectors. Med Hypotheses 68 (2007) 328-331
53. Uherek C., Fominaya J., and Wels W. A modular DNA carrier protein based on the structure of diphtheria toxin mediates target cell-specific gene delivery. J Biol Chem 273 (1998) 8835-8841
54. Vaysse L., Harbottle R., Bigger B., Bergau A., Tolmachov O., and Coutelle C. Development of a self-assembling nuclear targeting vector system based on the tetracycline repressor protein. J Biol Chem 279 (2004) 5555-5564
55. Vijayanathan V., Thomas T., and Thomas T.J. DNA nanoparticles and development of DNA delivery vehicles for gene therapy. Biochemistry 41 (2002) 14085-14094
56. Wadhwa M.S., Collard W.T., Adami R.C., McKenzie D.L., and Rice K.G. Peptide-mediated gene delivery: influence of peptide structure on gene expression. Bioconjug Chem 8 (1997) 81-88
57. Wagner E., Ogris M., and Zauner W. Polylysine-based transfection systems utilizing receptor-mediated delivery. Adv Drug Deliv Rev 30 (1998) 97-113
58. Wender P.A., Mitchell D.J., Pattabiraman K., Pelkey E.T., Steinman L., and Rothbard J.B. The design, synthesis, and evaluation of molecules that enable or enhance cellular uptake: peptoid molecular transporters. Proc Natl Acad Sci U S A 97 (2000) 13003-13008
59. Wolfert M.A., and Seymour L.W. Chloroquine and amphipathic peptide helices show synergistic transfection in vitro. Gene Ther 5 (1998) 409-414
60. Wu G.Y., and Wu C.H. Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. J Biol Chem 262 (1987) 4429-4432
61. Wu G.Y., and Wu C.H. Evidence for targeted gene delivery to Hep G2 hepatoma cells in vitro. Biochemistry 27 (1988) 887-892
62. Wu G.Y., and Wu C.H. Receptor-mediated gene delivery and expression in vivo. J Biol Chem 263 (1988) 14621-14624
63. Zabner J., Fasbender A.J., Moninger T., Poellinger K.A., and Welsh M.J. Cellular and molecular barriers to gene transfer by a cationic lipid. J Biol Chem 270 (1995) 18997-19007