Asymmetric 1-alkyl-2-acyl phosphatidylcholine: A helper lipid for enhanced non-viral gene delivery

International Journal of Pharmaceutics

Volumn 427, Issue 1, 2012, Pages 64-70

  Huang, Zhaohua ^a,b   Li, Weijun ^a,c   Szoka Jr , Francis C ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b MERRIMACK PHARMACEUTICALS   (United States)

^c BAYER CORPORATION   (United States)

Author keywords

Endosome escape; Liposome; Nanoparticles; Phospholipids; Transfection

Indexed keywords

ACYL GROUP; ALIPHATIC COMPOUND; ALKYL GROUP; FUNCTIONAL GROUP; GLYCEROL; LIPOPLEX; MACROGOL; NANOPARTICLE; PHOSPHATIDYLCHOLINE; PHOSPHORYLCHOLINE; UNCLASSIFIED DRUG;

ARTICLE; CHEMICAL STRUCTURE; CYTOTOXICITY; DRUG DESIGN; GENETIC TRANSFECTION; IN VITRO STUDY; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; SYNTHESIS;

ANIMALS; CATIONS; CELL LINE, TUMOR; ENDOSOMES; FATTY ACIDS, MONOUNSATURATED; GENE THERAPY; LIPOSOMES; MELANOMA, EXPERIMENTAL; MICE; NANOPARTICLES; PARTICLE SIZE; PHOSPHATIDYLCHOLINES; POLYETHYLENE GLYCOLS; TRANSFECTION;

EID: 84858749647     PISSN: 03785173     EISSN: 18733476     Source Type: Journal    
DOI: 10.1016/j.ijpharm.2011.06.022     Document Type: Article

References (41)

1. S. Audouy, and D. Hoekstra Cationic lipid-mediated transfection in vitro and in vivo (review) Mol. Membr. Biol. 18 2001 129 143 (Pubitemid 32666792)
2. S.A. Audouy, L.F. de Leij, D. Hoekstra, and G. Molema In vivo characteristics of cationic liposomes as delivery vectors for gene therapy Pharm. Res. 19 2002 1599 1605 (Pubitemid 35305510)
3. V. Chandrashekhar, M. Srujan, R. Prabhakar, R.C. Reddy, B. Sreedhar, K.K. Rentam, S. Kanjilal, and A. Chaudhuri Cationic amphiphiles with Fatty acyl chain asymmetry of coconut oil deliver genes selectively to mouse lung Bioconjug. Chem. 22 2011 497 509
4. V. Cherezov, H. Qiu, V. Pector, M. Vandenbranden, J.M. Ruysschaert, and M. Caffrey Biophysical and transfection studies of the diC(14)-amidine/DNA complex Biophys. J. 82 2002 3105 3117 (Pubitemid 34547654)
5. H. Eliyahu, N. Servel, A.J. Domb, and Y. Barenholz Lipoplex-induced hemagglutination: potential involvement in intravenous gene delivery Gene Ther. 9 2002 850 858 (Pubitemid 34716329)
6. A. ElOuahabi, M. Thiry, V. Pector, R. Fuks, J.M. Ruysschaert, and M. Vandenbranden The role of endosome destabilizing activity in the gene transfer process mediated by cationic lipids Feb Letters 414 1997 187 192 (Pubitemid 27389360)
7. A. Fasbender, J. Marshall, T.O. Moninger, T. Grunst, S. Cheng, and M.J. Welsh Effect of co-lipids in enhancing cationic lipid-mediated gene transfer in vitro and in vivo Gene Ther. 4 1997 716 725 (Pubitemid 27343134)
8. P.L. Felgner, T.R. Gadek, M. Holm, R. Roman, H.W. Chan, M. Wenz, J.P. Northrop, G.M. Ringold, and M. Danielsen Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure Proc. Natl. Acad. Sci. U. S. A. 84 1987 7413 7417
9. X. Gao, K.S. Kim, and D. Liu Nonviral gene delivery: what we know and what is next AAPS J. 9 2007 E92 104
10. J. Gaucheron, C. Boulanger, C. Santaella, N. Sbirrazzuoli, O. Boussif, and P. Vierling In vitro cationic lipid-mediated gene delivery with fluorinated glycerophosphoethanolamine helper lipids Bioconjug. Chem. 12 2001 949 963 (Pubitemid 33136865)
11. X. Guo, and F.C. Szoka Jr. Chemical approaches to triggerable lipid vesicles for drug and gene delivery Acc. Chem. Res. 36 2003 335 341
12. I.M. Hafez, N. Maurer, and P.R. Cullis On the mechanism whereby cationic lipids promote intracellular delivery of polynucleic acids Gene Ther. 8 2001 1188 1196 (Pubitemid 32776393)
13. M.E. Hayes, D.C. Drummond, K. Hong, J.W. Park, J.D. Marks, and D.B. Kirpotin Assembly of nucleic acid-lipid nanoparticles from aqueous-organic monophases Biochim. Biophys. Acta 1758 2006 429 442 (Pubitemid 43767730)
14. Z.H. Huang, W.J. Li, J.A. MacKay, and F.C. Szoka Thiocholesterol-based lipids for ordered assembly of bioresponsive gene carriers Mol. Ther. 11 2005 409 417 (Pubitemid 40277317)
15. S.W. Hui, M. Langner, Y.L. Zhao, P. Ross, E. Hurley, and K. Chan The role of helper lipids in cationic liposome-mediated gene transfer Biophys. J. 71 1996 590 599 (Pubitemid 26289143)
16. I. Koltover, T. Salditt, J.O. Radler, and C.R. Safinya An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery Science 281 1998 78 81 (Pubitemid 28354046)
17. R. Koynova, B. Tenchov, L. Wang, and R.C. Macdonald Hydrophobic moiety of cationic lipids strongly modulates their transfection activity Mol. Pharm. 6 2009 951 958
18. T. Kurosaki, T. Kitahara, M. Teshima, K. Nishida, J. Nakamura, M. Nakashima, H. To, H. Hukuchi, T. Hamamoto, and H. Sasaki Exploitation of De Novo helper-lipids for effective gene delivery J. Pharm. Pharm. Sci. 11 2008 56 67
19. W. Li, and F.C. Szoka Jr. Lipid-based nanoparticles for nucleic acid delivery Pharm. Res. 24 2007 438 449 (Pubitemid 46295166)
20. W.J. Li, Z.H. Huang, J.A. MacKay, S. Grube, and F.C. Szoka Low-pH-sensitive poly(ethylene glycol) (PEG)-stabilized plasmid nanlipoparticles: effects of PEG chain length, lipid composition and assembly conditions on gene delivery J. Gene Med. 7 2005 67 79
21. D.X. Liu, T. Ren, and X. Gao Cationic transfection lipids Curr. Med. Chem. 10 2003 1307 1315 (Pubitemid 36763938)
22. Y. Liu, L.C. Mounkes, H.D. Liggitt, C.S. Brown, I. Solodin, T.D. Heath, and R.J. Debs Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery Nat. Biotechnol. 15 1997 167 173 (Pubitemid 27081784)
23. S.E. McNeil, A. Vangala, V.W. Bramwell, P.J. Hanson, and Y. Perrie Lipoplexes formulation and optimisation: in vitro transfection studies reveal no correlation with in vivo vaccination studies Curr. Drug Deliv. 7 2010 175 187
24. M.H. Nantz, C.W. Dicus, B. Hilliard, S. Yellayi, S. Zou, and J.G. Hecker The benefit of hydrophobic domain asymmetry on the efficacy of transfection as measured by in vivo imaging Mol. Pharm. 7 2010 786 794
25. F. Paltauf, and A. Hermetter Preparation of alkyl ether and vinyl ether substrates for phospholipases Method Enzymol. 197 1991 134 149
26. F. Paltauf, and A. Hermetter Strategies for the synthesis of glycerophospholipids Prog. Lipid Res. 33 1994 239 328 (Pubitemid 24164724)
27. C.A. Prata, Y. Li, D. Luo, T.J. McIntosh, P. Barthelemy, and M.W. Grinstaff A new helper phospholipid for gene delivery Chem. Commun. (Camb) 2008 1566 1568 (Pubitemid 351423057)
28. C.R. Safinya, and I. Koltover Self-assembled structures of lipid/DNA nonviral gene delivery systems from synchrotron X-ray diffraction L. Huang, M.C. Hung, E. Wagner, Nonviral Vectors for Gene Therapy 1999 Academic Press San Diego 106 110
29. C.R. Safinya, E.B. Sirota, D. Roux, and G.S. Smith Universality in interacting membranes: the effect of cosurfactants on the interfacial rigidity Phys. Rev. Lett. 1989 1134
30. F. Sakurai, T. Nishioka, H. Saito, T. Baba, A. Okuda, O. Matsumoto, T. Taga, F. Yamashita, Y. Takakura, and M. Hashida Interaction between DNA-cationic liposome complexes and erythrocytes is an important factor in systemic gene transfer via the intravenous route in mice: the role of the neutral helper lipid Gene Ther. 8 2001 677 686 (Pubitemid 32496429)
31. F. Sakurai, T. Nishioka, F. Yamashita, Y. Takakura, and M. Hashida Effects of erythrocytes and serum proteins on lung accumulation of lipoplexes containing cholesterol or DOPE as a helper lipid in the single-pass rat lung perfusion system Eur. J. Pharm. Biopharm. 52 2001 165 172 (Pubitemid 32755895)
32. M.G. Stanton, and S.L. Colletti Medicinal chemistry of siRNA delivery J. Med. Chem. 53 2010 7887 7901
33. B.M. Tandia, C. Lonez, M. Vandenbranden, J.M. Ruysschaert, and A. Elouahabi Lipid mixing between lipoplexes and plasma lipoproteins is a major barrier for intravenous transfection mediated by cationic lipids J. Biol. Chem. 280 2005 12255 12261
34. A.S. Ulrich Biophysical aspects of using liposomes as delivery vehicles Bioscience Rep. 22 2002 129 150 (Pubitemid 35222729)
35. L.S. Uyechi, L. Gagne, G. Thurston, and F.C. Szoka Jr. Mechanism of lipoplex gene delivery in mouse lung: binding and internalization of fluorescent lipid and DNA components Gene Ther. 8 2001 828 836 (Pubitemid 32577187)
36. L. Wang, and R.C. MacDonald New strategy for transfection: mixtures of medium-chain and long-chain cationic lipids synergistically enhance transfection Gene Ther. 11 2004 1358 1362 (Pubitemid 39214001)
37. L. Xu, and T.J. Anchordoquy Cholesterol domains in cationic lipid/DNA complexes improve transfection Biochim. Biophys. Acta 1778 2008 2177 2181
38. Y. Xu, and F.C. Szoka Jr. Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection Biochemistry 35 1996 5616 5623 (Pubitemid 26143656)
39. R. Zeisig, A. Ress, I. Fichtner, and W. Walther Lipoplexes with alkylphospholipid as new helper lipid for efficient in vitro and in vivo gene transfer in tumor therapy Cancer Gene Ther. 10 2003 302 311 (Pubitemid 36423112)
40. Y. Zhang, E.L. Bradshaw-Pierce, A. Delille, D.L. Gustafson, and T.J. Anchordoquy In vivo comparative study of lipid/DNA complexes with different in vitro serum stability: effects on biodistribution and tumor accumulation J. Pharm. Sci. 97 2008 237 250
41. I.S. Zuhorn, V. Oberle, W.H. Visser, J.B. Engberts, U. Bakowsky, E. Polushkin, and D. Hoekstra Phase behavior of cationic amphiphiles and their mixtures with helper lipid influences lipoplex shape DNA translocation, and transfection efficiency Biophys. J. 83 2002 2096 2108