Novel series of non-glycerol-based cationic transfection lipids for use in liposomal gene delivery

Journal of Medicinal Chemistry

Volumn 42, Issue 21, 1999, Pages 4292-4299

  Banerjee, Rajkumar ^a   Prasanta Kumar, Das ^a   Srilakshmi, Gollapudi Venkata ^a   Chaudhuri, Arabinda ^a   Nalam Madhusudhana, Rao ^b  

^a INDIAN INSTITUTE OF CHEMICAL TECHNOLOGY   (India)

^b CENTRE FOR CELLULAR AND MOLECULAR BIOLOGY   (India)

Author keywords

[No Author keywords available]

Indexed keywords

LIPID TRANSFER PROTEIN; LIPOFECTAMINE; LIPOSOME; N,N DI N HEXADECYL N,N DIHYDROXYETHYLAMMONIUM BROMIDE:CHOLESTEROL:N N HEXADECYL N,N DIHYDROXYETHYLAMMONIUM BROMIDE; REAGENT; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; CATION EXCHANGE; CELL STRAIN COS1; DNA DRUG COMPLEX; GENE TARGETING; HYDROGEN BOND; LIPID ANALYSIS; MEMBRANE RECONSTITUTION; MOLECULAR INTERACTION; NONHUMAN; STRUCTURE ANALYSIS; SYNTHESIS;

ANIMALS; BETA-GALACTOSIDASE; CELL SURVIVAL; COS CELLS; ETHANOLAMINES; GENE TRANSFER TECHNIQUES; GENES, REPORTER; LIPIDS; PLASMIDS; QUATERNARY AMMONIUM COMPOUNDS; TRANSFECTION;

EID: 0345426296     PISSN: 00222623     EISSN: None     Source Type: Journal    
DOI: 10.1021/jm9806446     Document Type: Article

References (33)

1. (a) Indian and U.S. Patents on this work have been filed. Indian Patent Application No. 3324/DEL/98, 3325/DEL/98, and 3327/ DEL/98; all filed on Nov 9, 1998. U.S. Patent Application No. still waiting. (b) Abbreviations used: DOTMA, 1,2-dioleyl-3-N,N,N-trimethylaminopropane chloride; DC-Chol, 3-β-[N-(N′,N′-dimethylethyl)carbamoyl]cholesterol; DMDHP, (±)-N,N-[bis(2-hydroxyethyl)]-W-[2,3-bis(tetradecanoyloxy)propyl]ammonium chloride; DMRIE, 1,2-dimyristyloxypropyl-3-dimethylhydroxy-ethylammonium bromide; DOTAP, 1,2-dioleoyloxy-3-(trimethylamino)propane; DHDEAB, N,N-di-n-hexadecyl-N,N-dihydroxy-ethylammonium bromide; HDEAB, N-N-hexadecyl-N,N-dihydroxyethylammonium bromide; MOOHAC, N-methyl-N-n-octadecyl-N-oleyl-N-hydroxyethylammonium chloride; DOMHAC, N-methyl-N,N-di-n-octadecyl-N-hydroxyethylammonium chloride; DOHEMAB, N,N-di[O-hexadecanoyl]hydroxyethyl-N-hydroxyethyl-N-methylammonium bromide; DOSPA, 2,3-dioleyloxy-N-[2-(sperminecarboxamido)ethyl]N,N-dimethyl-1-propanamminium trifluoroacetate; DDAB, dioctadecyldimethylammonium bromide; DOPE, 1,2-dioleoylpropyl-3-phosphatidylethanolamine.
2. Miller, A. D. Human Gene Therapy Comes of Age. Nature 1992, 357, 455-460.
3. Dunbar, C.; Kohn, D.; Karlsson, S.; Barton, N.; Brady, R.; Cottler-Fox, M.; Crooks, G.; Emmons, R.; Esplin, J.; Leitman, S.; Lenarsky, C.; Nolta, J.; Parkman, R.; Pensiero, M.; Schifmann, R.; Tolstoshev, P.; Weinberg, K. Retroviral Mediated Transfer of the cDNA for Human Glucocerebrosidase into Hematopoietic Stem Cells of Patients with Gaucher Disease. A Phase I Study. Hum. Gene Ther. 1996, 7, 231-253.
4. Engelhardt, J. F.; Yang, Y.; Stratford-Perricaudet, L. D.; Allen, E. D.; Kozarsky, K.; Perricaudet, M.; Yankaskas, J. R.; Wilson, J. M. Direct Gene Transfer of Human CFTR into Human Bronchial Epithelia of Xenografts with EI-deleted Adenoviruses. Nature Genet. 1993, 4, 27-34.
5. Felgner, P. L. Particulate Systems and Polymers for in vitro and in vivo Delivery of Polynucleotides. Adv. Drug Delivery Rev. 1990, 5, 163-187.
6. Behr, J. P. Gene Transfer with Synthetic Cationic Amphiphiles: Prospects for Gene Therapy. Bioconjugate Chem. 1994, 5, 382-389.
7. Wyman, T. B.; Nicol, F.; Zelphati, O.; Scaria, P. V.; Plank, C.; Szoka, F. C., Jr. Design, Synthesis and Characterization of a Cationic Peptide that Binds to Nucleic Acids and Permeabilizes Bilayers. Biochemistry 1997, 36, 3008-3017.
8. Feigner, P; L.; Gadek, T. R.; Holm, M.; Roman, R.; Chan, W.; Wenz, M.; Northorp, J. P.; Ringold, G. M.; Danielsen, M. Lipofection: A Highly Efficient Lipid-Mediated DNA Transfection Procedure. Proc. Natl. Acad. Sci. U.S.A. 1987, 84, 7413-7417.
9. Zhu, N.; Liggitt, D.; Liu, Y.; Debs, R. Systemic Gene Expression after Intravenous DNA Delivery into Adult Mice. Science 1993, 261, 209-211.
10. Camerol, F. H.; Moghddam, M. J.; Bender, V. J.; Whittaker, R. G.; Mott, M.; Lockett, T. J. A Transfection Compound Series based on a Versatile Tris Linkage. Biochim. Biophys. Acta 1999, 1417, 37-50.
11. Floch, V.; Bolc'h, G. Le.; Gable-Guillaume, C.; Bris, N. Le.; Yaouanc, J.-J.; Abbayes, H. Des.; Fe'rec, C.; Cle'ment, J.-C. Phospholipids as Non-Viral Vectors for Gene Therapy. Eur. J. Med. Chem. 1998, 33, 923-934.
12. Miller, A. D. Cationic Liposomes in Gene Therapy. Angew. Chem., Int. Ed. Engl. 1998, 37, 1968-1785.
13. Wang, J.; Guo, X.; Xu, Y.; Barron, L.; Szoka, F. C., Jr. Synthesis and Characterization of Long Chain Alkyl Acyl Carnitine Esters. Potentially Biodegradable Cationic Lipids for use in Gene Delivery. J. Med. Chem. 1998, 41, 2207-2215.
14. Tan, F.; Hughes, J. A. Introduction of a Disulfide Bond into a Cationic Lipid Enhances Transgene Expression of Plasmid DNA. Biochem. Biophys. Res. Commun. 1998, 242, 141-145.
15. Hara, T.; Tan, Y.; Huang, L. In Vivo Gene Delivery to the Liver using Reconstituted Chylomicron Remnants as a Novel Nonviral Vector. Proc. Natl. Acad. Sci. U.S.A. 1997, 94, 14547-14552.
16. Templeton, N. S.; Lasic, D. D.; Frederik, P. M.; Strey, H. H.; Roberts, D. D.; Pavlakis, G. N. Improved DNA:Liposome Complexes for increased Systemic Delivery and Gene Expression. Nature Biotechnol. 1997, 15, 647-652.
17. Liu, Y.; Mounkes, L. C.; Liggitt, H. D.; Brown, C. S.; Solodin, I.; Heath, T. D.; Debs, R. J. Factors influencing the Efficiency of Cationic Liposome-Mediated Intravenous Gene Delivery. Nature Biotechnol. 1997, 15, 167-173.
18. Bennett, M. J.; Aberle, A. M.; Balasubramanium, R. P.; Malone, J. G.; Malone, R. W.; Nantz, M. H. Cationic Lipid-Mediated Gene Delivery to Murine Lung: Correlation of Lipid Hydration with in Vivo Transfection Activity. J. Med. Chem. 1997, 269, 4069-4078.
19. Hofland, H. E. J.; Shephard, L.; Sullivan, S. M. Formation of Stable Cationic Lipid/DNA Complexes for Gene Transfer. Proc. Natl. Acad. Sci. U.S.A. 1996, 93, 7305-7309.
20. Wheeler, C. J.; Felgner, P. L.; Tsai, Y. J.; Marshall, J.; Sukhu, L.; Doh, G.; Hartikka, J.; Nietupski, J.; Manthorpe, M.; Nichols, M.; Piewe, M.; Liang, X.; Norman, J.; Smith, A.; Cheng, S. H. A Novel Cationic Lipid Greatly Enhances Plasmid DNA Delivery and Expression in Mouse Lung. Proc. Natl. Acad. Sci. U.S.A. 1996, 93, 11454-11459.
21. Nantz, M. H.; Bennet, M. J.; Malone, R. W. Preparation of Bis-(acyloxy)alkanediaminium Compounds as Cationic Transport Reagents. U.S. Patent No. 5,527,928, 1996.
22. Caplen, N. J.; Alton, E. W.; Middleton, P. G.; Dorin, J. R.; Stevension, B. J.; Gao, X.; Durham, S. R.; Jeffery, P. K.; Hodson, M. E.; Coutelle, C.; Huang, L.; Porteous, D. J.; Williamson, R.; Geddes, D. M. Liposome-Mediated CFTR Gene Transfer to the Nasal Epithelium of Patients with Cystic Fibrosis. Nature Med. 1995, 1, 39-46.
23. Solodin, I.; Brown, C.; Bruno, M.; Chow, C.; Jang, E.-H.; Debs, R.; Heath, T. A Novel Series of Amphiphilic Imidazolinium Compounds for In Vitro and In Vivo Gene Delivery. Biochemistry 1995, 34, 13537-13544.
24. Liu, Y.; Liggitt, D.; Tu, G.; Zhong, W.; Gaensler, K.; Debs, R. Cationic Liposome-Mediated Intravenous Gene Delivery in Mice. J. Biol. Chem. 1995, 270, 24864-24870.
25. Felgner, J. H.; Kumar, R.; Sridhar, C. N.; Wheeler, C. J.; Tsai, Y.-J.; Border, R.; Ramsey, P.; Martin, M.; Feigner, P. L. Enhanced Gene Delivery and Mechanism Studies with a Novel Series of Cationic Lipid Formulations. J. Biol. Chem. 1994, 269, 2550-2561.
26. Alton, E. W.; Middleton, P. G.; Caplen, N. J.; Smith, S. N.; Steel, D. M.; Munkonge, F. M.; Jeffery, P. K.; Stevension, B. J.; McLachlan, G.; Dorin, J. R.; Porteous, D. J. Non-invasive Liposome-Mediated Gene Delivery Can Correct the Ion-Transport Defect in Cystic Fibrosis Mutant Mice. Nature Genet. 1993, 5, 135-142.
27. Stewart, M. J.; Plautz, G. E.; Del Buono, L.; Yang, Z. Y.; Xu, L.; Gao, X.; Huang, L.; Nabel, E. G.; Nabel, G. J. Gene Transfer In Vivo with DNA-Liposome Complexes: Safety and Acute Toxicity in Mice. Hum. Gene Ther. 1992, 3, 267-275.
28. Gao, X.; Huang, L. A Novel Cationic Liposome Reagent for Efficient Transfection of Mammalian Cells. Biochem. Biophys. Res. Commun. 1991, 179, 280-285.
29. Leventis, R.; Silvius, J. R. Interactions of Mammalian Cells with Lipid Dispersions containing Novel Metabolizable Cationic Amphiphiles. Biochim. Biophys. Acta 1990, 1023, 124-132.
30. Hansen, M. B.; Neilsen, S. E.; Berg, K. Re-examination and further Development of a Precise and Rapid Dye Method for measuring Cell Growth/Cell Kill. J. Immunol Methods 1989, 779, 203-210.
31. Tundo, P.; Kippenberger, D. J.; Klahn, P. L.; Prieto, N. E.; Jao, T.-C.; Fendler, J. H. J. Am. Chem. Soc. 1982, 104, 456-461.
32. Sambrook, J.; Fritsch, E. F.; Maniatis, T. Molecular Cloning; A Laboratory Manual, 2nd ed.; Cold Spring Harbor Laboratory Press: Cold Spring Harbor, NY, 1989.
33. Nantz, M. H.; Li, L.; Zhu, J.; Aho-Sharon, K. L.; Lim, D.; Erickson, K. L. Inductive Electron Withdrawal from Ammoniumm Ion Headgroups of Cationic Lipids and the Influence on DNA Transfection. Biochim. Biophys. Acta 1998, 1394, 219-223.