Design, preparation and application of nucleic acid delivery carriers

Biotechnology Advances

Volumn 32, Issue 4, 2014, Pages 804-817

  Yang, Jun ^a   Liu, Hongmei ^a,b   Zhang, Xin ^a  

^a INSTITUTE OF PROCESS ENGINEERING   (China)

^b UNIVERSITY OF CHINESE ACADEMY OF SCIENCES   (China)

Author keywords

Dendrimersome; Gene delivery; Liposome; Non viral vectors; Polymersome

Indexed keywords

BIOMOLECULES; GENE THERAPY; GENE TRANSFER; LIPOSOMES; NUCLEIC ACIDS; VIRUSES;

DENDRIMERSOME; GENE DELIVERY; GENE DELIVERY VECTORS; NON-VIRAL CARRIERS; NONVIRAL VECTORS; NUCLEIC ACID DELIVERIES; POLYMERSOMES; TRANSFECTION EFFICIENCY;

VECTORS;

CANCER; DESIGN; DISEASE TREATMENT; GENETIC ENGINEERING; NUCLEIC ACID; VIRAL DISEASE; VIRUS;

DNA; LIPID; LIPOSOME; NUCLEIC ACID; POLYETHYLENEIMINE; POLYMER; SMALL INTERFERING RNA;

ANIMAL; BIOMIMETICS; CHEMISTRY; DRUG DELIVERY SYSTEM; GENE THERAPY; GENE TRANSFER; GENE VECTOR; HEPG2 CELL LINE; HUMAN; METABOLISM; MOUSE; NANOMEDICINE; NANOTECHNOLOGY; PROCEDURES; TUMOR CELL LINE;

ANIMALS; BIOMIMETICS; CELL LINE, TUMOR; DNA; DRUG DELIVERY SYSTEMS; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HEP G2 CELLS; HUMANS; LIPIDS; LIPOSOMES; MICE; NANOMEDICINE; NANOTECHNOLOGY; NUCLEIC ACIDS; POLYETHYLENEIMINE; POLYMERS; RNA, SMALL INTERFERING;

EID: 84901979146     PISSN: 07349750     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.biotechadv.2013.11.004     Document Type: Review

References (81)

1. Akinc A., Thomas M., Klibanov A.M., Langer R. Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis. J Gene Med 2005, 7:657-663.
2. Armstrong J.K., Hempel G., Koling S., Chan L.S., Fisher T., Meiselman H.J., et al. Antibody against poly(ethylene glycol) adversely affects PEG-asparaginase therapy in acute lymphoblastic leukemia patients. Cancer Res 2007, 110:103-111.
3. Boussif O., Lezoualc'h F., Zanta M.A., Mergny M.D., Scherman D., Demeneix B., et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci U S A 1995, 92:7297-7301.
4. Buhleier E., Wehner W., Vögtle F. 'Cascade'- and 'nonskid-chain-like' syntheses of molecular cavity topologies. Synthesis 1978, 2:155-158.
5. Caminade A.-M., Laurent R., Delavaux-Nicot B., Majoral J.-P. "Janus" denderimers: syntheses and properties. New J Chem 2012, 36:217-226.
6. Check E. Gene therapy put on hold as third child develops cancer. Nature 2005, 433:561.
7. Chen Y., Huang L. Tumor-targeted delivery of siRNA by non-viral vector: safe and effective cancer therapy. Expert Opin Drug Deliv 2008, 5:1301-1311.
8. Choi Y.H., Liu F., Kim J.-S., Choi Y.K., Park J.S., Kim S.W. Polyethylene glycol-grafted poly-L-lysine as polymeric gene carrier. J Control Release 1998, 54:39-48.
9. Chowdhury E.H. Nuclear targeting of viral and non-viral DNA. Expert Opin Drug Deliv 2009, 6:697-703.
10. Collins S.A., B-a Guinn, Harrison P.T., Scallan M.F., O'Sullivan G.C., Tangney M. Viral vectors in cancer immunotherapy: which vector for which strategy?. Curr Gene Ther 2008, 8:66-78.
11. Cooray S., Howe S.J., Thrasher A.J. Retrovirus and lentivirus vector design and methods of cell conditioning. Methods Enzymol 2012, 507:29-57.
12. Dai J., Zou S., Pe Y., Cheng D., Ai H., Shuai X. Polyethylenimine-grafted copolymer of poly(L-lysine) and poly(ethylene glycol) for gene delivery. Biomaterials 2011, 32:1694-1705.
13. Dai Z., Gjetting T., Mattebjerg M.A., Wu C., Andresen T.L. Elucidating the interplay between DNA-condensing and free polycations in gene transfection through a mechanistic study of linear and branched PEI. Biomaterials 2011, 32:8626-8634.
14. Davis M.E., Chen Z., Shin D.M. Nanoparticle therapeutics: an emerging treatment modality for cancer. Nat Rev Drug Discov 2008, 7:771-782.
15. Dean M., Fojo T., Bates S. Tumor stem cells and drug resistance. Nat Rev Cancer 2005, 5:275-284.
16. Deng R., Yue Y., Jin F., Chen Y., Kung H.-F., Lin M.C.M., et al. Revisit the complexation of PEI and DNA - How to make low cytotoxic and highly efficient PEI gene transfection non-viral vectors with a controllable chain length and structure?. J Control Release 2009, 140:40-46.
17. Ellis J. Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum Gene Ther 2005, 16:1241-1246.
18. Ewert K.K., Evans H.M., Ahmad A., Slack N.L., Lin A.J., Martin-Herranz A., et al. Lipoplex structures and their distinct cellular pathways. Adv Genet 2005, 53:119-155.
19. Ewert K.K., Evans H.M., Bouxsein N.F., Safinya C.R. Dendritic cationic lipids with highly charged headgroups for efficient gene delivery. Bioconjug Chem 2006, 17:877-888.
20. Ewert K.K., Evans H.M., Zidovska A., Bouxsein N.F., Ahmad A., Safinya C.R. A columnar phase of dendritic lipid-based cationic complexes for gene delivery: hexagonally ordered embedded in a DNA honeycomb lattice. J Am Chem Soc 2006, 128:3998-4006.
21. Ewert K.K., Zidovska A., Ahmad A., Bouxsein N.F., Evans H.M., Mcallister C.S., et al. Cationic liposome-nucleic acid complexes for gene delivery and silencing: pathways and mechanisms for plasmid DNA and siRNA. Top Curr Chem 2010, 296:191-226.
22. Felgner P.L., Gadek T.R., Holm M., Roman R., Chan H.W., Wenz M., et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci U S A 1987, 84:7413-7417.
23. Fischer D., Bieber T., Li Y., Elsässer H.-P., Kissel T. A novel non-viral vector for DNA delivery based on low molecular weight, branched polyethylenimine: effect of molecular weight on transfection efficiency and cytotoxicity. Pharm Res 1999, 16:1273-1279.
24. Gary D.J., Puri N., Won Y.-Y. Polymer-based siRNA delivery: perspectives on the fundamental and phenomenological distinctions from polymer-based DNA delivery. J Control Release 2007, 121:64-73.
25. Gomes-da-Silva L.C., Fonseca N.A., Moura V., MCPd Lima, Simões S., Moreira J.N. Lipid-based nanoparticles for siRNA delivery in cancer therapy: paradigms and challenges. Acc Chem Res 2012, 45:1163-1171.
26. Green J.J., Zugates G.T., Tedford N.C., Huang Y.-H., Griffith L.G., Lauffenburger D.A., et al. Combinatorial modification of degradable polymers enables transfection of human cells comparable to adenovirus. Adv Mater 2007, 19:2836-2842.
27. Green J.J., Langer R., Anderson D.G. A combinatorial polymer library approach yields insight into nonviral gene delivery. Acc Chem Res 2008, 41:749-759.
28. Guo X., Huang L. Recent advances in nonviral vectors for gene delivery. Acc Chem Res 2012, 45:971-979.
29. Hartman Z.C., Appledorn D.M., Amalfitano A. Adenovirus vector induced innate immune responses: impact upon efficacy and toxicity in gene therapy and vaccine applications. Virus Res 2008, 132:1-14.
30. Hatakeyama H., Akita H., Kogure K., Oishi M., Nagasaki Y., Kihira Y., et al. Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid. Gene Ther 2007, 14:68-77.
31. Hatakeyama H., Akita H., Harashima H. A multifunctional envelope type nano device (MEND) for gene delivery to tumors based on the EPR effect: a strategy for overcoming the PEG dilemma. Adv Drug Deliv Rev 2011, 63:152-160.
32. Hofman J., Buncek M., Haluza R., Streinz Ludvik, Ledvina M., Cigler P. In vitro transfection mediated by dendrigraft poly(L-lysines): the effect of structure and molecule size. Macromol Biosci 2013, 13:167-176.
33. Hong C.A., Kim J.S., Lee S.H., Kong W.H., Park T.G., Mok H., et al. Reductively dissociable siRNA-polymer hybrid nanogels for efficient targeted gene silencing. Adv Funct Mater 2013, 23:316-322.
34. Huang R., Liu S., Shao K., Han L., Ke W., Liu Y., et al. Evaluation and mechanism studies of PEGylated dendrigraft poly-L-lysines as novel gene delivery vectors. Nanotechnology 2010, 21:265101-265111.
35. Huang B., Kukowska-Latallo J.F., Tang S., Zong H., Johnson K.B., Desai A., et al. The facile synthesis of multifunctional PAMAM dendrimer conjugates through copper-free click chemistry. Bioorg Med Chem Lett 2012, 22:3152-3156.
36. Ishida T., Masuda K., Ichikawa T., Ichihara M., Irimura K., Kiwada H. Accelerated clearance of a second injection of PEGylated liposomes in mice. Int J Pharm 2003, 255:167-174.
37. Iyer A.K., Khaled G., Fang J., Maeda H. Exploiting the enhanced permeability and retention effect for tumor targeting. Drug Discov Today 2006, 11:812-818.
38. Kawabata K., Takakura Y., Hashida M. The fate of plasmid DNA after intravenous injection in mice: involvement of scavenger receptors in its hepatic uptake. Pharm Res 1995, 12:825-830.
39. Kogure K., Moriguchi R., Sasaki K., Ueno M., Futaki S., Harashima H. Development of a non-viral multifunctional envelope-type nano device by a novel lipid film hydration method. J Control Release 2004, 98:317-323.
40. Koltover I., Salditt T., Rädler J.O., Safinya C.R. An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery. Science 1998, 281:78-81.
41. Lehrman S. Virus treatment questioned after gene therapy death. Nature 1999, 401:517-518.
42. Li J., Loh X.J. Cyclodextrin-based supramolecular architectures: syntheses, structures, and applications for drug and gene delivery. Adv Drug Deliv Rev 2008, 60:1000-1017.
43. Li L., Cole J., Margolin D.A. Cancer stem cell and stromal microenvironment. Ochsner J 2013, 13:109-118.
44. Lin P.J.C., Tam Y.Y.C., Hafez I., Sandhu A., Chen S., Ciufolini M.A., et al. Influence of cationic lipid composition on uptake and intracellular processing of lipid nanoparticle formulations of siRNA. Nanomedicine 2013, 9:233-246.
45. Liu Y., Huang L. Designer lipids advance systemic siRNA delivery. Mol Ther 2010, 18:669-670.
46. Liu G., Molas M., Grossmann G.A., Pasumarthy M., Perales J.C., Cooper M.J., et al. Biological properties of poly-L-lysine-DNA complexes generated by cooperative binding of the polycation. J Biol Chem 2001, 276:34379-34387.
47. Lynn D.M., Langer R. Degradable poly(β-amino esters): synthesis, characterization, and self-assembly with plasmid DNA. J Am Chem Soc 2000, 122:10761-10768.
48. Maeda H., Fang J., Inutsuka T., Kitamoto Y. Vascular permeability enhancement in solid tumor: various factors, mechanisms involved and its implications. Int Immunopharmacol 2003, 3:319-328.
49. Männistö 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 2002, 83:169-182.
50. Marshall E. Gene therapy death prompts review of adenovirus vector. Science 1999, 286:2244-2245.
51. Martello F., Piest M., Engbersen J.F.J., Ferruti P. Effects of branched or linear architecture of bioreducible poly(amido amine)s on their in vitro gene delivery properties. J Control Release 2012, 164:372-379.
52. Mével M., Kamaly N., Carmona S., Oliver M.H., Jorgensen M.R., Crowther C., et al. DODAG; a versatile new cationic lipid that mediates efficient delivery of pDNA and siRNA. J Control Release 2010, 143:222-232.
53. Mintzer M.A., Simanek E.E. Nonviral vectors for gene delivery. Chem Rev 2009, 109:259-302.
54. Namgung R., Kim J., Singha K., Kim C.H., Kim W.J. Synergistic effect of low cytotoxic linear polyethylenimine and multiarm polyethylene glycol: study of physicochemical properties and in vitro gene transfection. Mol Pharm 2009, 6:1826-1835.
55. Ohsaki M., Okuda T., Wada A., Hirayama T., Niidome T., Aoyagi H. In vitro gene transfection using dendritic poly(L-lysine). Bioconjug Chem 2002, 13:510-517.
56. Patnaik S., Gupta K.C. Novel polyethylenimine-derived nanoparticles for in vivo gene delivery. Expert Opin Drug Deliv 2013, 10:215-228.
57. Percec V., Wilson D.A., Leowanawat P., Wilson C.J., Hughes A.D., Kaucher M.S., et al. Self-assembly of janus dendrimer into uniform dendrimersomes and other complex architectures. Science 2010, 328:1009-1014.
58. Peterca M., Percec V., Leowanawat P., Bertin A. Predicting the size and properties of dendrimersomes from the lamellar structure of their amphiphilic Janus dendrimers. J Am Chem Soc 2011, 133:20507-20520.
59. Sakurai Y., Hatakeyama H., Sato Y., Hyodo M., Akita H., Harashima H. Gene silencing via RNAi and siRNA quantification in tumor tissue using MEND, a liposomal siRNA delivery system. Mol Ther 2013, 21:1195-1203.
60. Saranya N., Moorthi A., Saravanan S., Devi M.P., Selvamurugan N. Chitosan and its derivatives for gene delivery. Int J Biol Macromol 2011, 48:234-238.
61. Sato Y., Hatakeyama H., Sakurai Y., Hyodo M., Akita H., Harashima H. A pH-sensitive cationic lipid facilitates the delivery of liposomal siRNA and gene silencing activity in vitro and in vivo. J Control Release 2012, 163:267-276.
62. Selkirk S.M. Gene therapy in clinical medicine. Postgrad Med J 2004, 80:560-570.
63. Semple S.C., Akinc A., Chen J., Sandhu A.P., Mui B.L., Cho C.K., et al. Rational design of cationic lipids for siRNA delivery. Nat Biotechnol 2010, 28:172-176.
64. Shen Y., Tang H., Zhan Y., Kirk E.A.V., Murdoch W.J. Degradable poly(β-amino ester) nanoparticles for cancer cytoplasmic drug delivery. Nanomedicine 2009, 5:192-201.
65. Soltanian S., Matin M.M. Cancer stem cells and cancer therapy. Tumor Biol 2011, 32:425-440.
66. Son S., Namgung R., Kim J., Singha K., Kim W.J. Bioreducible polymers for gene silencing and delivery. Acc Chem Res 2012, 45:1100-1112.
67. Soutschek J., Akinc A., Bramlage B., Charisse K., Constien R., Donoghue M., et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 2004, 432:173-178.
68. Takakura Y., Nishikawa M., Yamashita F., Hashida M. Development of gene drug delivery systems based on pharmacokinetic studies. Eur J Pharm Sci 2001, 13:71-76.
69. Torchilin V. Tumor delivery of macromolecular drugs based on the EPR effect. Adv Drug Deliv Rev 2011, 63:131-135.
70. Wightman L., Kircheis R., Rössler V., Carotta S., Ruzicka R., Kursa M., et al. Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo. J Gene Med 2001, 3:362-372.
71. Yamagata M., Kawano T., Shiba K., Mori T., Katayama Y., Niidome T. Structural advantage of dendritic poly(l-lysine) for gene delivery into cells. Bioorg Med Chem Lett 2007, 15:526-532.
72. Yang X., Dou S., Sun T., Mao C., Wang H., Wang J. Systemic delivery of siRNA with cationic lipid assisted PEG-PLA nanoparticles for cancer therapy. J Control Release 2011, 156:203-211.
73. Yang X.-Z., Dou S., Wang Y.-C., Long H.-Y., Xiong M.-H., Mao C.-Q., et al. Single-step assembly of cationic lipid-polymer hybrid nanoparticles for systemic delivery of siRNA. ACS Nano 2012, 6:4955-4965.
74. Yue Y., Wu C. Progress and perspectives in developing polymeric vectors for in vitro gene delivery. Biomater Sci 2013, 1:152-170.
75. Yue Y., Jin F., Deng R., Cai J., Chen Y., Lin M.C.M., et al. Revisit complexation between DNA and polyethylenimine - effect of uncomplexed chains free in the solution mixture on gene transfection. J Control Release 2011, 155:67-76.
76. Yue Y., Jin F., Deng R., Cai J., Dai Z., Lin M.C.M., et al. Revisit complexation between DNA and polyethylenimine - effect of length of free polycationic chains on gene transfection. J Control Release 2011, 152:143-151.
77. Zhang X., Sharma K.K., Boeglin M., Ogier J., Mainard D., Voegel J.-C., et al. Transfection ability and intracellular DNA pathway of nanostructured gene-delivery systems. Nano Lett 2008, 8:2432-2436.
78. Zhang X., Oulad-Abdelghani M., Zelkin A.N., Wang Y., Haîkel Y., Mainard D., et al. Poly(l-lysine) nanostructured particles for gene delivery and hormone stimulation. Biomaterials 2010, 31:1699-1706.
79. Zhang B., Ma X., Murdoch W., Radosz M., Shen Y. Bioreducible poly(amido amine)s with different branching degrees as gene delivery vectors. Biotechnol Bioeng 2013, 110:990-998.
80. Zhong Z., Feijen J., Lok M.C., Hennink W.E., Christensen L.V., Yockman J.W., et al. Low molecular weight linear polyethylenimine-b-poly(ethylene glycol)-b-polyethylenimine triblock copolymers: synthesis, characterization, and in vitro gene transfer properties. Biomacromolecules 2005, 6:3440-3448.
81. Zugates G.T., Peng W., Zumbuehl A., Jhunjhunwala S., Huang Y.-H., Langer R., et al. Rapid optimization of gene delivery by parallel end-modification of poly(β-amino ester)s. Mol Ther 2007, 15:1306-1312.