Polymers for gene delivery across length scales

Nature Materials

Volumn 5, Issue 6, 2006, Pages 439-451

  Putnam, David ^a  

^a Cornell University   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CHEMICAL COMPOUNDS; DISEASES; MOLECULAR STRUCTURE; OPTIMIZATION; PATIENT TREATMENT; PH EFFECTS; POLYACRYLATES; POLYETHYLENE GLYCOLS; POLYMERS; VECTOR QUANTIZATION; VIRUSES;

GENE DELIVERY; GENE THERAPY; POLY N-METHACRYLOXYSUCCINAMIDE; POLYMER-BASED VECTOR DESIGN;

GENE TRANSFER;

DNA; POLYMER;

CHEMISTRY; GENE THERAPY; GENE TRANSFER; GENE VECTOR; HUMAN; INSTRUMENTATION; METHODOLOGY; NANOTECHNOLOGY; REVIEW;

DNA; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; NANOTECHNOLOGY; POLYMERS;

EID: 33744805790     PISSN: 14761122     EISSN: 14764660     Source Type: Journal    
DOI: 10.1038/nmat1645     Document Type: Review

References (105)

1. Luo, D. & Saltzman, W. M. Synthetic DNA delivery systems. Nature Biotechnol. 18, 33-37 (2000).
2. Schaffer, D. V. & Lauffenburger, D. A. Targeted synthetic gene delivery vectors. Curr. Opin, Mol. Ther. 2, 155-161 (2000).
3. Mahato, R. I., Smith, L. C. & Rolland A. Pharmaceutical perspectives of nonviral gene therapy. Adv. Genet. 41, 95-156 (1999).
4. Demeneix, B., Hassani Z. & Behr, J. P. Towards multifunctional synthetic vectors Curr. Gene Ther. 4, 445-455 (2004).
5. Roth, C. M. & Sundaram, S. Engineering synthetic vectors for improved DNA delivery: insights from intracellular pathways. Annu. Rev. Biomed. Eng. 6, 397-126 (2004).
6. Mahato, R. I. Non-viral peptide-based approaches to gene delivery J. Drug Target. 7, 249-268 (1999).
7. Schatzlein, A. G. Non-viral vectors in cancer gene therapy: principles and progress. Anticancer Drugs 12, 275-304 (2001).
8. Wiethoff, C. M. & Middaugh, C. R. Barriers to nonviral gene delivery J. Pharm. Sci. 92, 203-217 (2003).
9. Cho, Y. W., Kim, J. D. & Park, K. Polycation gene delivery systems: escape from endosomes to cytosol. J. Pharm. Pharmacol. 55, 1-734 (2003).
10. Pannier, A. K. & Shea, L. D. Controlled release systems for DNA delivery. Mol. Ther. 10, 19-26 (2001).
11. Molas, M. et al. Receptor-mediated gene transfer vectors: progress towards genetic pharmaceuticals. Curr. Gene Ther. 3, 468-485 (2003).
12. Roy, I. & Gupta, M. N. Smart polymeric materials: emerging biochemical applications. Chem. Biol. 10, 1161-1171 (2003).
13. Kakizawa, Y. & Kataoka, K. Block copolymer micelles for delivery of gene and related compounds. Adv. Drug Deliv. Rev. 54, 203-222 (2002).
14. Tang, M. X. & Szoka, F. C. The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther. 4, 823-832 (1997).
15. Dubruel, P. et al. Synthetic polyamines as vectors for gene delivery. Polym. Int. 51, 948-957 (2002).
16. Merdan, T., Kopecek, J. & Kissel, T. Prospects for cationic polymers in gene and oligonucleotide therapy against cancer. Adv. Drug Deliv. Rev. 54, 715-758 (2002).
17. Murphy, J. E. et al. A combinatorial approach to the discovery of efficient cationic peptoid reagents for gene therapy. Proc. Natl Acad. Sci USA 95, 1517-1522 (1998).
18. Lenssen, K., Jantscheff, P., von Kiedrowski, G. & Massing, U. Combinatorial synthesis of new cationic lipids and high-throughput screening of their transfection properties. ChemBioChem 3, 852-858 (2002).
19. Lynn, D. M., Anderson, D. G., Putnam, D. & Langer, R. Accelerated discovery of synthetic transfection vectors: parallel synthesis and screening of a degradable polymer library. J. Am. Chem. Soc. 123, 8155-8156 (2001).
20. Anderson, D. G., Lynn, D. M. & Langer, R. Semi-automated synthesis and screening of a large library of degradable cationic polymers for gene delivery. Angew. Chem. Int. Edn 42, 3153-3158 (2003).
21. Akinc, A., Lynn, D. M., Anderson, D. G. & Langer. R. Parallel synthesis and biophysical characterization of a degradable polymer library for gene delivery. J. Am. Chem. Soc. 125, 5316-5323 (2003).
22. Akinc, A., Anderson, D. G., Lynn, D. M. & Langer, R. Synthesis of poly(β-amino ester)s optimized for highly effective gene delivery, Bioconjug. Chem. 14, 979-988 (2003).
23. Anderson, D. G. et al. A polymer library approach to suicide gene therapy for cancer. Proc. Natl Acad. Sci. USA 101, 16028-16033 (2004).
24. Mammen, M., Dahmann, G. & Whitesides, G. Effective inhibitors of hemagglutination by influenze virus synthesized from polymers having active ester groups. Insight in mechanism of inhibition. J. Med. Chem. 38, 4179-4190 (1995).
25. Godwin, A., Hartenstein, M., Müller, A. & Brocchini, S. Synthesis of a polymeric precursor by ATRP for conversion to polymer-drug conjugates. Am. Chem. Soc. Polym. Chem. Div. 41,1002-1003 (2000).
26. Godwin, A., Hartenstein, M., Müller, A. & Brocchini, S. Narrow molecular weight distribution precursors for polymer-drug conjugates. Angew. Chem. Int. Edn 40, 594-597 (2001).
27. Pedone, E., Li, X., Koseva, N., Alpar, O. & Brocchini, S. An information rich biomedical polymer library. J. Mater. Chem. 13, 2825-2837 (2003).
28. Batz, H., Franzmann, G. & Ringsdorf, H. Model reactions for synthesis of pharmacologically active polymers byway of monomeric and polymeric reactive esters. Angew Chem Int. Edn Engl. 11, 1103-1104 (1972).
29. Putnam, D. & Kopecek, J. Polymer conjugates with anticancer activity. Adv. Polymer Sci. 122, 55-123 (1995).
30. Ferruti P., Betellini, A. & Fere, A. High polymers of acrylic and methacrylic esters of N-hydroxysuccinimide as polyacrylamide and polymethacrylamide precursors. Polymer 13, 462-464 (1972).
31. Davis, F. F. The origin of pegnology. Adv. Drug. Del. Rev. 54, 457-458 (2002).
32. MacKay, J. A., Deen, D. F. & Szoka, F. C. Jr Distribution in brain of liposomes after convection enhanced delivery; modulation by particle charge, particle diameter, and presence of steric coating. Brain Res 1035, 139-153 (2005).
33. Tsubery, H., Mironchik, M., Fridkin, M. & Shechter, Y. Prolonging the action of protein and peptide drugs by a novel approach of reversible polyethylene glycol modification. J. Biol. Chem. 279, 38118-38121 (2004).
34. Alemany, R., Suzuki, K. & Curiel, D. T. Blood clearance rates of adenovirus type 5 in mice. J. Gen. Virol. 81, 2605-2609 (2000).
35. Chillon, M., Lee, J. H., Fasbender, A. & Welsh, M. I. Adenovirus complexed with polyethylene glycol and cationic lipid is shielded from neutralizing antibodies in vitro. Gene Ther. 5, 995-1002 (1998).
36. O'Riordan, C. R. et al. PEGylation of adenovirus with retention of infectivity and protection from neutralizing antibody in vitro and in vivo. Hum. Gene Ther. 10, 1349-1358 (1999).
37. Croyle, M. A., Yu, Q.-C. & Wilson, J. M. Development of a rapid method for the PEGylation of adenoviruses with enhanced transduction and improved stability under harsh storage conditions. Hum. Gene Ther. 11, 1713-1722 (2000).).
38. Green, N. K. et al. Extended plasma circulation time and decreased toxicity of polymer-coated adenovirus. Gene Ther. 11, 1256-1263 (2004).
39. Wickham, T. J., Roolvink, P., Brough, D. E. & Kovesdi, I. Adenovirus targeted to heparin-containing receptors increases its gene delivery efficiency to multiple cell types. Nature Biotechnol. 14, 1570-1573 (1996).
40. Wickham, T. J. et al. Increased in vitro and in vivo gene transfer by adenovirus vectors containing chimeric fiber proteins. J. Virol. 71, 8221-8229 (1997).
41. Reynolds, P., Dmitriev, I. & Curiel, D. Insertion of an RGD motif into the HI loop of adenovirus fiber protein alters the distribution of transgene expression of the systemically administered vector. Gene Ther. 6, 1336-1339 (1999).
42. Lanciotti, J. et. al. Targeting adenoviral vectors using heterofunctional polyethylene glycol FGF2 conjugates. Mol. Ther. 8, 99-107 (2003).
43. Fisher, K. D. et al. Polymer-coated adenovirus permits efficient retargeting and evades neutralising antibodies. Gene Ther. 8, 341-348 (2001).
44. Lee, G. K., Maheshri, N., Kaspar, B. & Schaffer, D. V. PEG conjugation moderately protects adeno-associated viral vectors against antibody neutralization. Biotechnol. Bioeng. 92, 24-34 (2005).
45. Croyle, M. A. et al. PEGylation of a vesicular stomatitis virus G pseudotyped lentivirus vector prevents inactivation in serum. J. Virol. 78, 912-921 (2004).
46. Soong, N. W. et al. Molecular breeding of viruses Nature Genet 25, 436-439 (2000).
47. Maheshri, N., Koerber, J. T., Kaspar, B. & Schaffer, D. V. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nature Biotechnol. 24, 198-204 (2006).
48. Donnelly, J. J., Wahren, B. & Liu, M. A. DNA vaccines: progress and challenges. J. Immunol. 175, 633-639 (2005).
49. Hedley, M. L., Curley, J. & Urban, R. Microspheres containing plasmid encoded antigens elicit cytotoxic T-cell responses. Nature Med. 4, 365-368 (1998).
50. Jones, D. H., Corris, S., McDonald, S., Clegg, J. C. & Farrar, G. H. Poly(D,L-lactide-co-glycolide)-encapsulated plasmid DNA elicits systemic and mucosal antibody responses to encoded protein after oral administration. Vaccine 15, 814-817 (1997).
51. Tabata, I. & Ikada, Y. Phagocytosis of polymer microspheres by macrophages. Adv. Polym. Sci. 94, 107-141 (1990).
52. Luby, T. M. et al. Repeated immunization with plasmid DNA formulated in poly(lactide-co-glycolide) micropartides is well tolerated and stimulates durable T cell responses to the tumor associated antigen cytochrome P450 1B1. Clin. Immunol. 112, 45-53 (2004).
53. Crum, C. P. et al. Dynamics of human papillomavirus infection between biopsy and excision of cervical intraepithelial neoplasia: results from the ZYC101a protocol. J. Infect. Dis. 189, 1348-1354 (2004).
54. Fu, K., Pack, D. W., Klibanov, A. M. & Langer, R. Visual evidence of acidic environment within degrading poly(lactic-co-glycolic acid) (PLGA) microspheres. Pharm. Res. 17, 100-106 (2000).
55. Wang, D., Robinson, D. R., Kwon, G. S. & Samuel, J. Encapsulation of plasmid DNA in biodegradable poly(D,L-lactic-co-glycolic acid) microspheres as a novel approach for immunogene delivery. J. Control. Release 57, 9-18 (1999).
56. Kaech, S. M. & Ahmed, R. Memory CD8+ T cell differentiation: initial antigen encounter triggers a development program in naïve cells. Nature Immunol. 2, 415-422 (2001).
57. Princiotta, M. F. et al. Quantitating protein synthesis, degradation and endogenous antigen processing. Immunity 18, 343-354 (2003).
58. Ando, S., Putnam, D., Pack, D. W. & Langer, R. PLGA microspheres containing plasmid DNA: preservation of supercoiled DNA via cryopreparation and carbohydrate stabilization. J. Pharm. Sci. 88, 126-130 (1999).
59. Singh, M., Briones, M., Ott, G. & O'Hagan, D. Cationic microparticles: A potent delivery system for DNA vaccines. Proc. Natl Acad. Sci. USA 97, 811-816 (2000).
60. O'Hagan D. T., Singh, M. Ulmer J. B. Microparticles for the Delivery of DNA Vaccines. Immunol. Rev. 199, 191-200 (2004).
61. Heller, J., Barr, J., Ng, S. Y., Schwach-Abdellaoui, K. & Gurny, R. Poly(ortho esters): synthesis, characterization, properties and uses. Adv. Drug Deliv. Rev. 54, 1015-1039 (2002).
62. Cordes, E. H. & Bull, H. G. Mechanism and catalysis for hydrolysis of acetals, ketals, and ortho esters. Chem. Rev. 74, 581-604 (1974).
63. Wang, C. et al. Molecularly engineered poly(ortho ester) microspheres for enhanced delivery of DNA vaccines. Nature Mater. 3, 190-196 (2004).
64. Akinc, A., Anderson, D. G., Lynn, D. M. & Langer, R. Synthesis of poly(beta-amino ester)s optimized for highly effective gene delivery. Bioconjug. Chem. 14, 979-88 (2003).
65. Lynn, D. M. & Langer R. Degradable Poly(β-amino esters): synthesis, characterization, and self-assembly with plasmid DNA. J. Am. Chem. 122, 10761-10768 (2000).
66. Lynn, D. M., Amiji, M. M. & Langer, R. pH Responsive polymer microspheres: rapid release of encapsulated material within the range of intracellular pH. Angew. Chem. Int. Edn. 40, 1707-1710 (2001).
67. Little, S. R. et al. Poly(β-amino esters)-containing microparticles enhance the activity of nonviral geneitic vaccines. Proc. Natl Acad. Sci. USA 101, 9534-9539 (2004).
68. Oster, C. G. et al. Cationic microparticles consisting of poly(lactide-co-glycolide) and polyethylenimine as carriers systems for parental DNA vaccination. J. Control. Release 104, 359-377 (2005).
69. Beer, S. J. et al. Poly (lactlc-glycolic) acid copolymer encapsulation of recombinant adenovirus reduces immunogenicity in vivo. Gene Ther. 5, 740-746 (1998).
70. Langer, R. & Tirrell, D. A. Designing materials for biology and medicine. Nature 428, 487-492 (2004).
71. Niklason, L. E. & Langer, R. Prospects for organ and tissue replacement. J. Am. Med. Assoc. 285, 573-576 (2001).
72. Langer, R. New methods of drug delivery. Science 249, 1527-1533 (1992).
73. Shea, L. D., Smiley, E., Bonadio, J. & Mooney, D. J. DNA delivery from polymer matrices for tissue engineering. Nature Biotechnol. 17, 551-554 (1999).
74. Kong, H. J. et al. Non-viral gene delivery regulated by stiffness of cell adhesion substrates. Nature Mater. 4, 460-464 (2005).
75. Ziauddin, J. & Sabatini, D. M. Microarrays of cells expressing defined cDNAs. Nature 411, 107-110 (2001).
76. Truong-Le, V. L. et al. Gene transfer by DNA-gelatin nanospheres. Arch. Biochem. Biophys. 361, 47-56 (1999).
77. Leong, K. W. et al. DNA-polycation nanospheres as non-viral gene delivery vehicles. J. Control. Release 53, 183-193 (1998).
78. Zhang, J., Chua, L. S. & Lynn, D. M. Multilayered thin films that sustain the release of functional DNA under physiological conditions. Langmuir 20, 8015-8021 (2004).
79. Shen, H., Tan, J. & Saltzman, W. M. Surface-mediated gene transfer from nanocomposites of controlled texture. Nature Mater. 3, 569-574 (2004).
80. Segura, T. & Shea, L. D. Surface tethered DNA complexes for enhanced gene delivery, Bioconjugate. Chem. 13, 621-629 (2002).
81. Segura, T., Volk, M. J. & Shea, L. D. Substrate-mediated DNA delivery: role of the cationic polymer structure and extent of modification. J. Control. Rel. 93, 69-84 (2003).
82. Huang, Y.-C., Riddle, K., Rice, K. G. & Mooney, D. J. Long-term in vivo gene expression via delivery of PEI-DNA condensates from porous polymer scaffolds. Human Gene Ther. 16, 609-617 (2005).
83. Huang, Y.-C., Simmons, C., Kaigler, D., Rice, K. J. & Mooney, D. J. Bone regeneration in a rat cranial defect with delivery of PEI-condensed plasmid DNA encoding for bone morphogenetic protein 4 (BMP-4). Gene Ther. 12, 418-426 (2005).
84. Cohen-Sacks, H. et al. Delivery and expression of pDNA embedded in collagen matrices. J. Control. Rel. 95, 309-320 (2004).
85. Scherer, F., Ulrike, S., Putz, U., Stemberger, A. & Plank, C. Nonviral vector loaded collagen sponges for sustained gene delivery in vitro and in vivo. J. Gene Med. 4, 634-643 (2002).
86. Haider, M., Megeed, Z. & Ghandehari, H. Genetically engineered polymers: status and prospects for controlled release. J. Control. Rel. 95, 1-26 (2004).
87. Herweijer, H. et al. Time course of gene expression after plasmid DNA gene transfer to the liver. J. Gene Med. 3, 280-291 (2001).
88. Miao, C. H. A novel gene expression system: non-viral gene transfer for hemophilia as model systems. Adv. Genet. 54, 143-177 (2005).
89. Ivics, Z., Hackett, P. B., Plasterk, R. H. & Izsvak, Z. Molecular reconstruction of Sleeping Beauty, a Tcl-like transposon from fish, and its transposition in human cells. Cell 91, 501-510 (1997).
90. Hackett, P. B., Ekker, S. C., Largaespada, D. A. & McIvor, R. S. Sleeping Beauty transposon-mediated gene therapy for prolonged expression. Adv. Genet. 54, 189-232 (2005).
91. Ginsburg, D. S. & Calos, M. P. Site-specific integration with φC31 integrase for prolonged expression of therapeutic genes. Adv. Genet. 54, 179-187 (2005).
92. http://www.wiley.co.uk/genetherapy/clinical
93. http://www.mgipharma.com
94. Otten, G. R. et al. Enhanced potency of plasmid DNA microparticle human immunodeficiency virus vaccines in rhesus macaques by using a priming-boosting regimen with recombinant proteins. J. Virol. 79, 8189-8200 (2005).
95. Elbashir, S. M. et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411, 494-498 (2001).
96. Hammond, S. M., Bernstein, E., Beach, D. & Hannon, G. J. An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophilia cells. Nature 404, 293-296 (2000).
97. Zamore, P., Tuschl, T., Sharp, P. & Bartel, D. RNAi: Double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell 101, 25-33 (2000).
98. Oishi, M., Nagasaki, Y., Itaka, K., Nishiyama, N., & Kataoka, K. Lactosylated poly(ethylene glycol)-siRNA conjugate through acid-labile beta-thiopropionate linkage to construct pH sensitive polyion complex micelles achieving enhanced gene silencing in hepatoma cells. I Am. Chem. Soc. 127, 1624-5 (2005).
99. Kim, S. H., Jeong, J. H., Cho, K. C., Kim, S. W. & Park, T. G. Target-specific gene silencing by siRNA plasmid DNA complexed with folate-modified poly(ethylenimine). J. Control. Rel. 104, 223-232 (2005).
100. Soutschek, J. et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432, 173-178 (2004).
101. Chen, L. & Deem, M. W. Monte Carlo methods for small molecule high-throughput experimentation. J. Chem. Inf. Comput Sci. 41, 950-957 (2001).
102. Maheshri, N. & Schaffer, D. V. Computational and experimental analysis of DNA shuffling. Proc. Natl Acad. Sci. USA 100, 3071-3076 (2003).
103. Brocchini, S., James, K., Tangpasuthadol, V. & Kohn, J. Structure-property correlations in a combinatorial library of degradable biomaterials. J. Biomed. Mater. Res. 42, 66-75 (1998).
104. Brocchini, S., James, K., Tangpasuthadol, V. & Kbhn, J. A combinatorial approach for polymer design. J. Am. Chem. Soc. 119, 4553-4554 (1997).
105. Anderson, D. G., Putnam, D., Lavik, E. B., Mahmood, T. A. & Langer, R. Biomaterial microarrays: rapid, microscale screening of polymer cell interaction Biomater. 26, 4892-4897 (2005).