Novel cationic cardiolipin analogue-based liposome for efficient DNA and small interfering RNA delivery in vitro and in vivo

Cancer Gene Therapy

Volumn 12, Issue 3, 2005, Pages 321-328

  Chien, Pei Yu ^a   Wang, Jinkang ^a   Carbonaro, Danielle ^a   Lei, Sabrina ^a   Miller, Bruce ^a   Sheikh, Saifuddin ^a   Ali, Shoukath M ^a   Ahmad, Moghis U ^a   Ahmad, Imran ^a  

^a NeoPharm Inc   (United States)

Author keywords

Cationic cardiolipin analogue; Reporter gene; siRNA; Transfection; Xenograft tumor model

Indexed keywords

1,2 DIOLEOYL 3 TRIMETHYLAMMONIOPROPANE; C RAF PROTEIN; CARDIOLIPIN; CATIONIC CARDIOLIPIN DERIVATIVE; DNA; LIPOSOME; RAF PROTEIN; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; COMBINED IMMUNODEFICIENCY; CONTROLLED STUDY; GENE DELIVERY SYSTEM; GENETIC ASSOCIATION; GENETIC TRANSFECTION; GROWTH INHIBITION; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; MALE; MOUSE; NONHUMAN; NUCLEIC ACID ANALYSIS; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; REPORTER GENE; TUMOR GROWTH;

ANIMALS; BETA-GALACTOSIDASE; BODY WEIGHT; CARDIOLIPINS; CELL LINE, TUMOR; DNA; GENE THERAPY; HUMANS; LIPOSOMES; LUCIFERASES; MALE; MICE; MICE, INBRED BALB C; NEOPLASMS; PROTO-ONCOGENE PROTEINS C-RAF; RHODAMINES; RNA, SMALL INTERFERING; TRANSFECTION; TRANSPLANTATION, HETEROLOGOUS;

EID: 14644413531     PISSN: 09291903     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.cgt.7700793     Document Type: Article

References (33)

1. Felgner PL, Gadek TR, Holm M, et al. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA. 1987;84:7413-7417.
2. Audouy SA, de Leij LF, Hoekstra D, Molema G. In vivo characteristics of cationic liposomes as delivery vectors for gene therapy. Pharm Res. 2002;19:1599-1605.
3. Pedroso de Lima MC, Neves S, Filipe A, Duzgunes N, Simoes S. Cationic liposomes for gene delivery: from biophysics to biological applications. Curr Med Chem. 2003;10:1221-1231.
4. Barron LG, Uyechi LS, Szoka Jr FC. Cationic lipids are essential for gene delivery mediated by intravenous administration of lipoplexes. Gene Ther. 1999;6:1179-1183.
5. Barron LG, Gagne L, Szoka Jr FC. Lipoplex-mediated gene delivery to the lung occurs within 60 minutes of intravenous administration. Hum Gene Ther. 1999;10:1683-1694.
6. Krasnici S, Werner A, Eichhorn ME, et al. Effect of the surface charge of liposomes on their uptake by angiogenic tumor vessels. Int J Cancer. 2003;105:561-567.
7. Uchida E, Mizuguchi H, Ishii-Watabe A, Hayakawa T. Comparison of the efficiency and safety of non-viral vector-mediated gene transfer into a wide range of human cells. Biol Pharm Bull. 2002;25:891-897.
8. Loisel S, Floch V, Le Gall C, Ferec C. Factors influencing the efficiency of lipoplexes mediated gene transfer in lung after intravenous administration. J Liposome Res. 2001;11:127-138.
9. Eliyahu H, Servel N, Domb AJ, Barenholz Y. Lipoplex-induced hemagglutination: potential involvement in intravenous gene delivery. Gene Ther. 2002;9:850-858.
10. Loisel S, Le Gall C, Doucet L, Ferec C, Floch V. Contribution of plasmid DNA to hepatotoxicity after systemic administration of lipoplexes. Hum Gene Ther. 2001;12:685-696.
11. Tousignant JD, Gates AL, Ingram LK, et al. Comprehensive analysis of the acute toxicities induced by systemic administration of cationic lipid:plasmid DNA complexes in mice. Hum Gene Ther. 2000;11:2493-2513.
12. Zou Y, Zong G, Ling YH, Perez-Soler R. Development of cationic liposome formulations for intratracheal gene therapy of early lung cancer. Cancer Gene Ther. 2000;7:683-696.
13. Stopeck AT, Jones A, Hersh EM, et al. Phase II study of direct intralesional gene transfer of allovectin-7, an HLA-B7/beta2-microglobulin DNA-liposome complex, in patients with metastatic melanoma. Clin Cancer Res. 2001;7:2285-2291.
14. Noone PG, Hohneker KW, Zhou Z, et al. Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis. Mol Ther. 2000;1:105-114.
15. Hatch GM. Cardiolipin: biosynthesis, remodeling and trafficking in the heart and mammalian cells. Int J Mol Med. 1998;1:33-41.
16. Song YK, Liu F, Chu S, Liu D. Characterization of cationic liposome-mediated gene transfer in vivo by intravenous administration. Hum Gene Ther. 1997;8:1585-1594.
17. Gaucheron J, Wong T, Wong KF, Maurer N, Cullis PR. Synthesis and properties of novel tetraalkyl cationic lipids. Bioconjug Chem. 2002;13:671-675.
18. Kasireddy K, Ahmad MU, Ali SM, Ahmad I. Synthesis of novel cationic cardiolipin analogues for the optimal delivery of therapeutic agents. Tetrahedron Lett. 2004;45:2743-2746.
19. Caplen NJ, Mousses S. Short interfering RNA (siRNA)-mediated RNA interference (RNAi) in human cells. Ann NY Acad Sci. 2003;1002:56-62.
20. Woessmann W, Damm-Welk C, Fuchs U, Borkhardt A. RNA interference: new mechanisms for targeted treatment? Rev Clin Exp Hematol. 2003;7:270-291.
21. Lei S, Chien PY, Sheikh S, Zhang A, Ali S, Ahmad I. Enhanced therapeutic efficacy of a novel liposome-based formulation of SN-38 against human tumor models in SCID mice. Anticancer Drugs. 2004;15:773-778.
22. Skehan P, Storeng R, Scudiero D, et al. New colorimetric cytotoxicity assay for anticancer-drug screening. J Natl Cancer Inst. 1990;82:1107-1112.
23. Wang J, Guo X, Xu Y, Barron L, Szoka Jr FC. 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.
24. Almofti MR, Harashima H, Shinohara Y, Almofti A, Baba Y, Kiwada H. Cationic liposome-mediated gene delivery: biophysical study and mechanism of internalization. Arch Biochem Biophys. 2003;410:246-253.
25. Felgner JH, Kumar R, Sridhar CN, et al. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J Biol Chem. 1994;269:2550-2561.
26. Sakurai F, Nishioka T, Yamashita F, Takakura Y, Hashida M. 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. 2001;52:165-172.
27. McLean JW, Fox EA, Baluk P, et al. Organ-specific endothelial cell uptake of cationic liposome-DNA complexes in mice. Am J Physiol. 1997;273:H387-H404.
28. Anwer K, Kao G, Proctor B, Rolland A, Sullivan S. Optimization of cationic lipid/DNA complexes for systemic gene transfer to tumor lesions. J Drug Target. 2000;8:125-135.
29. Hofland HE, Shephard L, Sullivan SM. Formation of stable cationic lipid/DNA complexes for gene transfer. Proc Natl Acad Sci USA. 1996;93:7305-7309.
30. Kolch W. Meaningful relationships: the regulation of the Ras/raf/MEK/ERK pathway by protein interaction. Biochem J. 2000;351:289-305.
31. Storm SM, Brennscheidt U, Sithanandam G, Rapp UR. Raf oncogenes in carcinogenesis. Crit Rev Oncogen. 1990;2:1-8.
32. Monia BP, Sasmor H, Johnston JF, et al. Sequence-specific antitumor activity of a phosphorothioate oligodeoxyribonucleotide targeted to human C-raf kinase supports an antisense mechanism of action in vivo. Proc Natl Acad Sci USA. 1996;93:15481-15484.
33. Mewani RR, Tang W, Rahman A, et al. Enhanced therapeutic effects of doxorubicin and paclitaxel in combination with liposome-entrapped ends-modified raf antisense oligonucleotide against human prostate, lung and breast tumor models. Int J Oncol. 2004;24:1181-1188.