Combinatorial synthesis and high-throughput screening of alkyl amines for nonviral gene delivery

Bioconjugate Chemistry

Volumn 24, Issue 9, 2013, Pages 1543-1551

  Li, Linxian ^a,b   Wang, Fengjian ^b   Wu, Yihang ^a   Davidson, Gary ^a   Levkin, Pavel A ^a,b  

^a KARLSRUHE INSTITUTE OF TECHNOLOGY   (Germany)

^b UNIVERSITY OF HEIDELBERG   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

AMINES; EFFICIENCY; GENE TRANSFER; LIPIDS; STRUCTURAL OPTIMIZATION; SYNTHESIS (CHEMICAL);

ALKYLAMINE; BIOLOGICAL RESEARCH; CATIONIC LIPIDS; CELL-BE; CELL/B.E; CELL/BE; COMBINATORIAL SYNTHESIS; HIGH THROUGHPUT SCREENING; NON-VIRAL GENE DELIVERY; PLASMID DNA;

LIPOSOMES;

ALIPHATIC AMINE; LIPOFECTAMINE;

ALKYLATION; ARTICLE; ELECTROSPRAY MASS SPECTROMETRY; GENETIC TRANSFECTION; HIGH THROUGHPUT SCREENING; HYDROPHOBICITY; IN VITRO GENE TRANSFER; LIGHT SCATTERING; NONVIRAL GENE DELIVERY SYSTEM; PARTICLE SIZE; SURFACE CHARGE; SYNTHESIS;

ALKYLATION; AMINES; DNA; HEK293 CELLS; HUMANS; LIPIDS; TRANSFECTION;

EID: 84884391528     PISSN: 10431802     EISSN: 15204812     Source Type: Journal    
DOI: 10.1021/bc400158w     Document Type: Article

References (50)

1. Graham, F. L. and van der Eb, A. J. (1973) A new technique for the assay of infectivity of human adenovirus 5 DNA Virology 52, 456-467
2. Hofland, H. E., Shephard, L., and Sullivan, S. M. (1996) Formation of stable cationic lipid/DNA complexes for gene transfer Proc. Natl. Acad. Sci. U.S.A. 93, 7305-7309 (Pubitemid 26243545)
3. Wheeler, C. J., Felgner, P. L., Tsai, Y. J., Marshall, J., Sukhu, L., Doh, S. G., Hartikka, J., Nietupski, J., Manthorpe, M., Nichols, M., Plewe, M., Liang, X., Norman, J., Smith, A., and Cheng, S. H. (1996) A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung Proc. Natl. Acad. Sci. U.S.A. 93, 11454-11459 (Pubitemid 26347142)
4. Semple, S. C., Akinc, A., Chen, J., Sandhu, A. P., Mui, B. L., Cho, C. K., Sah, D. W., Stebbing, D., Crosley, E. J., Yaworski, E., Hafez, I. M., Dorkin, J. R., Qin, J., Lam, K., Rajeev, K. G., Wong, K. F., Jeffs, L. B., Nechev, L., Eisenhardt, M. L., Jayaraman, M., Kazem, M., Maier, M. A., Srinivasulu, M., Weinstein, M. J., Chen, Q., Alvarez, R., Barros, S. A., De, S., Klimuk, S. K., Borland, T., Kosovrasti, V., Cantley, W. L., Tam, Y. K., Manoharan, M., Ciufolini, M. A., Tracy, M. A., de Fougerolles, A., MacLachlan, I., Cullis, P. R., Madden, T. D., and Hope, M. J. (2010) Rational design of cationic lipids for siRNA delivery Nat. Biotechnol. 28, 172-176
5. Heyes, J., Palmer, L., Bremner, K., and MacLachlan, I. (2005) Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids J. Controlled Release 107, 276-287 (Pubitemid 41316290)
6. Zhi, D., Zhang, S., Wang, B., Zhao, Y., Yang, B., and Yu, S. (2010) Transfection efficiency of cationic lipids with different hydrophobic domains in gene delivery Bioconjugate Chem. 21, 563-577
7. Boussif, O., Lezoualch, F., Zanta, M. A., Mergny, M. D., Scherman, D., Demeneix, B., and Behr, J. P. (1995) A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine Proc. Natl. Acad. Sci. U.S.A. 92, 7297-7301
8. Godbey, W. T., Wu, K. K., and Mikos, A. G. (1999) Tracking the intracellular path of poly(ethylenimine)/DNA complexes for gene delivery Proc. Natl. Acad. Sci. U.S.A. 96, 5177-5181 (Pubitemid 29214556)
9. Ogris, M., Brunner, S., Schuller, S., Kircheis, R., and Wagner, E. (1999) PEGylated DNA/transferrin-PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery Gene Ther. 6, 595-605 (Pubitemid 29192668)
10. Kircheis, R., Kichler, A., Wallner, G., Kursa, M., Ogris, M., Felzmann, T., Buchberger, M., and Wagner, E. (1997) Coupling of cell-binding ligands to polyethylenimine for targeted gene delivery Gene Ther. 4, 409-418 (Pubitemid 27226583)
11. Wiseman, J. W., Goddard, C. A., McLelland, D., and Colledge, W. H. (2003) A comparison of linear and branched polyethylenimine (PEI) with DCChol/DOPE liposomes for gene delivery to epithelial cells in vitro and in vivo Gene Ther. 10, 1654-1662 (Pubitemid 37069493)
12. Wagner, E., Zatloukal, K., Cotten, M., Kirlappos, H., Mechtler, K., Curiel, D. T., and Birnstiel, M. L. (1992) Coupling of adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes Proc. Natl. Acad. Sci. U.S.A. 89, 6099-6103
13. Curiel, D. T., Agarwal, S., Wagner, E., and Cotten, M. (1991) Adenovirus enhancement of transferrin-polylysine-mediated gene delivery Proc. Natl. Acad. Sci. U.S.A. 88, 8850-8854 (Pubitemid 21915453)
14. KukowskaLatallo, J. F., Bielinska, A. U., Johnson, J., Spindler, R., Tomalia, D. A., and Baker, J. R. (1996) Efficient transfer of genetic material into mammalian cells using Starburst polyamidoamine dendrimers Proc. Natl. Acad. Sci. U.S.A. 93, 4897-4902 (Pubitemid 26163501)
15. Tang, M. X., Redemann, C. T., and Szoka, F. C. (1996) In vitro gene delivery by degraded polyamidoamine dendrimers Bioconjugate Chem. 7, 703-714 (Pubitemid 27012712)
16. Dufes, C., Uchegbu, I. F., and Schatzlein, A. G. (2005) Dendrimers in gene delivery Adv. Drug Delivery Rev. 57, 2177-2202 (Pubitemid 41759392)
17. Bielinska, A. U., Chen, C. L., Johnson, J., and Baker, J. R. (1999) DNA complexing with polyamidoamine dendrimers: Implications for transfection Bioconjugate Chem. 10, 843-850 (Pubitemid 29456373)
18. Zinselmeyer, B. H., Mackay, S. P., Schatzlein, A. G., and Uchegbu, I. F. (2002) The lower-generation polypropylenimine dendrimers are effective gene-transfer agents Pharm. Res. 19, 960-967 (Pubitemid 34808705)
19. Song, E. W., Zhu, P. C., Lee, S. K., Chowdhury, D., Kussman, S., Dykxhoorn, D. M., Feng, Y., Palliser, D., Weiner, D. B., Shankar, P., Marasco, W. A., and Lieberman, J. (2005) Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors Nat. Biotechnol. 23, 709-717 (Pubitemid 41716361)
20. McNaughton, B. R., Cronican, J. J., Thompson, D. B., and Liu, D. R. (2009) Mammalian cell penetration, siRNA transfection, and DNA transfection by supercharged proteins Proc. Natl. Acad. Sci. U.S.A. 106, 6111-6116
21. Kichler, A., Leborgne, C., Marz, J., Danos, O., and Bechinger, B. (2003) Histidine-rich amphipathic peptide antibiotics promote efficient delivery of DNA into mammalian cells Proc. Natl. Acad. Sci. U.S.A. 100, 1564-1568 (Pubitemid 36254487)
22. Lam, J. K. W., Liang, W., Lan, Y., Chaudhuri, P., Chow, M. Y. T., Witt, K., Kudsiova, L., and Mason, A. J. (2012) Effective endogenous gene silencing mediated by pH responsive peptides proceeds via multiple pathways J. Controlled Release 158, 293-303
23. Bonoiu, A. C., Mahajan, S. D., Ding, H., Roy, I., Yong, K. T., Kumar, R., Hu, R., Bergey, E. J., Schwartz, S. A., and Prasad, P. N. (2009) Nanotechnology approach for drug addiction therapy: Gene silencing using delivery of gold nanorod-siRNA nanoplex in dopaminergic neurons Proc. Natl. Acad. Sci. U.S.A. 106, 5546-5550
24. Chakravarthy, K. V., Bonoiu, A. C., Davis, W. G., Ranjan, P., Ding, H., Hu, R., Bowzard, J. B., Bergey, E. J., Katz, J. M., Knight, P. R., Sambhara, S., and Prasad, P. N. (2010) Gold nanorod delivery of an ssRNA immune activator inhibits pandemic H1N1 influenza viral replication Proc. Natl. Acad. Sci. U.S.A. 107, 10172-10177
25. Salem, A. K., Searson, P. C., and Leong, K. W. (2003) Multifunctional nanorods for gene delivery Nat. Mater. 2, 668-671
26. Scherer, F., Anton, M., Schillinger, U., Henkel, J., Bergemann, C., Kruger, A., Gansbacher, B., and Plank, C. (2002) Magnetofection: enhancing and targeting gene delivery by magnetic force in vitro and in vivo Gene Ther. 9, 102-109 (Pubitemid 34189525)
27. Yezhelyev, M. V., Qi, L. F., O'Regan, R. M., Nie, S., and Gao, X. H. (2008) Proton-sponge coated quantum dots for siRNA delivery and intracellular imaging J. Am. Chem. Soc. 130, 9006-9012 (Pubitemid 352000838)
28. Giljohann, D. A., Seferos, D. S., Prigodich, A. E., Patel, P. C., and Mirkin, C. A. (2009) Gene regulation with polyvalent siRNA-nanoparticle conjugates J. Am. Chem. Soc. 131, 2072-2073
29. Kay, M. A., Glorioso, J. C., and Naldini, L. (2001) Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics Nat. Med. 7, 33-40 (Pubitemid 32095639)
30. Zufferey, R., Nagy, D., Mandel, R. J., Naldini, L., and Trono, D. (1997) Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo Nat. Biotechnol. 15, 871-875
31. Xia, H. B., Mao, Q. W., Paulson, H. L., and Davidson, B. L. (2002) siRNA-mediated gene silencing in vitro and in vivo Nat. Biotechnol. 20, 1006-1010
32. Kafri, T., Blomer, U., Peterson, D. A., Gage, F. H., and Verma, I. M. (1997) Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors Nat. Genet. 17, 314-317 (Pubitemid 27475995)
33. Zufferey, R., Dull, T., Mandel, R. J., Bukovsky, A., Quiroz, D., Naldini, L., and Trono, D. (1998) Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery J. Virol. 72, 9873-9880 (Pubitemid 28520853)
34. Dull, T., Zufferey, R., Kelly, M., Mandel, R. J., Nguyen, M., Trono, D., and Naldini, L. (1998) A third-generation lentivirus vector with a conditional packaging system J. Virol. 72, 8463-8471 (Pubitemid 28471781)
35. Sheridan, C. (2011) Gene therapy finds its niche Nat. Biotechnol. 29, 121-128
36. Felgner, P. L., Gadek, T. R., Holm, M., Roman, R., Chan, H. W., Wenz, M., Northrop, J. P., Ringold, G. M., and Danielsen, M. (1987) Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure Proc. Natl. Acad. Sci. U.S.A. 84, 7413-7417
37. Walker, S., Sofia, M. J., Kakarla, R., Kogan, N. A., Wierichs, L., Longley, C. B., Bruker, K., Axelrod, H. R., Midha, S., Babu, S., and Kahne, D. (1996) Cationic facial amphiphiles: a promising class of transfection agents Proc. Natl. Acad. Sci. U.S.A. 93, 1585-1590 (Pubitemid 26075169)
38. Miller, A. D. (1998) Cationic liposomes for gene therapy Angew. Chem., Int. Ed. 37, 1769-1785 (Pubitemid 28397773)
39. Akinc, A., Zumbuehl, A., Goldberg, M., Leshchiner, E. S., Busini, V., Hossain, N., Bacallado, S. A., Nguyen, D. N., Fuller, J., Alvarez, R., Borodovsky, A., Borland, T., Constien, R., de Fougerolles, A., Dorkin, J. R., Jayaprakash, K. N., Jayaraman, M., John, M., Koteliansky, V., Manoharan, M., Nechev, L., Qin, J., Racie, T., Raitcheva, D., Rajeev, K. G., Sah, D. W. Y., Soutschek, J., Toudjarska, I., Vornlocher, H. P., Zimmermann, T. S., Langer, R., and Anderson, D. G. (2008) A combinatorial library of lipid-like materials for delivery of RNAi therapeutics Nat. Biotechnol. 26, 561-569 (Pubitemid 351668042)
40. Love, K. T., Mahon, K. P., Levins, C. G., Whitehead, K. A., Querbes, W., Dorkin, J. R., Qin, J., Cantley, W., Qin, L. L., Racie, T., Frank-Kamenetsky, M., Yip, K. N., Alvarez, R., Sah, D. W. Y., de Fougerolles, A., Fitzgerald, K., Koteliansky, V., Akinc, A., Langer, R., and Anderson, D. G. (2010) Lipid-like materials for low-dose, in vivo gene silencing Proc. Natl. Acad. Sci. U.S.A. 107, 1864-1869
41. Cho, S. W., Goldberg, M., Son, S. M., Xu, Q. B., Yang, F., Mei, Y., Bogatyrev, S., Langer, R., and Anderson, D. G. (2009) Lipid-like nanoparticles for small interfering RNA delivery to endothelial cells Adv. Funct. Mater. 19, 3112-3118
42. Mahon, K. P., Love, K. T., Whitehead, K. A., Qin, J., Akinc, A., Leshchiner, E., Leshchiner, I., Langer, R., and Anderson, D. G. (2010) Combinatorial approach to determine functional group effects on lipidoid-mediated siRNA delivery Bioconjugate Chem. 21, 1448-1454
43. Sun, S., Wang, M., Knupp, S. A., Soto-Feliciano, Y., Hu, X., Kaplan, D. L., Langer, R., Anderson, D. G., and Xu, Q. B. (2012) Combinatorial library of lipidoids for in vitro DNA delivery Bioconjugate Chem. 23, 135-140
44. Li, L., Zahner, D., Su, Y., Gruen, C., Davidson, G., and Levkin, P. A. (2012) A biomimetic lipid library for gene delivery through thiol-yne click chemistry Biomaterials 33, 8160-8166
45. Jones, T. R., Kang, I. H., Wheeler, D. B., Lindquist, R. A., Papallo, A., Sabatini, D. M., Golland, P., and Carpenter, A. E. (2008) CellProfiler Analyst: data exploration and analysis software for complex image-based screens BMC Bioinf. 9, 482
46. Chesnoy, S. and Huang, L. (2000) Structure and function of lipid-DNA complexes for gene delivery Annu. Rev. Biophys. Biomol. Struct. 29, 27-47 (Pubitemid 30599587)
47. Farhood, H., Serbina, N., and Huang, L. (1995) The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer Biochim. Biophys. Acta 1235, 289-295
48. Koynova, R., Wang, L., and MacDonald, R. C. (2006) An intracellular lamellar-nonlamellar phase transition rationalizes the superior performance of some cationic lipid transfection agents Proc. Natl. Acad. Sci. U.S.A. 103, 14373-14378 (Pubitemid 44484757)
49. Synatschke, C. V., Schallon, A., Jerome, V., Freitag, R., and Müller, A. H. (2011) Influence of polymer architecture and molecular weight of poly(2-(dimethylamino)ethyl methacrylate) polycations on transfection efficiency and cell viability in gene delivery Biomacromolecules 12, 4247-4255
50. Whitehead, K. A., Sahay, G., Li, G. Z., Love, K. T., Alabi, C. A., Ma, M., Zurenko, C., Querbes, W., Langer, R. S., and Anderson, D. G. (2011) Synergistic silencing: combinations of lipid-like materials for efficacious siRNA delivery Mol. Ther. 19, 1688-1694