Novel cationic lipids for gene delivery and gene therapy

Bibliography

• SMITH A: Viral vectors in gene therapy. Nature (1995)49:807–838.
   Google Scholar
• BOULIKAS T: Gene therapy to human diseases: ex vivo and in vivo studies. Intl Oncol. (1996) 9:1239–1251.
   Google Scholar
• FRENCH ANDERSON W: Human gene therapy. Nature (1998) 392:25–30.
   Google Scholar
• BISHOP JM: The molecular genetics of cancer. Science (1987) 235:305–311.
   PubMed Web of Science ®Google Scholar
• YANG Y, NUNES FA, BERENCSI K et al.: Cellular immu-nity to viral antigenes limits El-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA (1994) 91:4407–4411.
   PubMed Web of Science ®Google Scholar
• LEE SG, KIM S, KIM BG, ROBBINS PD: Optimization of environmental factors for the production and han-dling of recombinant retrovirus. Appl. Microbiol. Bio-technol (1996) 45(4):477–483.
   PubMed Web of Science ®Google Scholar
• STRAYER DS: Novel SV40 virus SVluc vector, luciferase reporter gene transfer to mouse, appl. gene therapy papova virus enzyme mammal animal expression. Cancer Gene Ther. (1995) 2(4):321.
   Google Scholar
• KRASNYKH VN, MIKHEEVA GV, DOUGLAS JT, CURIEL DT: Generation of recombinant adenovirus vectors with modified fibers for altering viral tropism. J. Vim]. (1996) 70(10)6839–6846.
   Google Scholar
• YANEZ RJ, PORTER ACG: Therapeutic gene targeting. Gene Ther. (1998) 5:149–159.
   PubMedGoogle Scholar
• SMITH KT, SHEPHERD AJ, BOYD JE, LEES GM: Gene deliv-ery systems for use in gene therapy: an overview of quality assurance and safety issues. Gene Ther. (1996) 3:190–200.
   PubMed Web of Science ®Google Scholar
• FELGNER PL, GADEK TG, HOLM M et al: Lipofection: ahighly efficient, lipid-mediated DNA-transfection pro-cedure. Proc. Natl. Acad. Sci. USA. (1987) 84:7413–7417.
   PubMed Web of Science ®Google Scholar
• BEHR JP, DEMENEIX B, LOEFFLER JP, PEREZ-MUTUL J: Ef-ficient gene transfer into mammalian primary endo-crine cells with lipopolyamine-coated DNA. Proc. Nati Acad. Sci. USA (1989) 86:6982–6986.
   PubMed Web of Science ®Google Scholar
• GAO X, HUANG L: A novel cationic liposome reagentfor efficient transfection of mammalian cells. Biochem. Biophys. Res. Commun. (1991) 179:280–285.
   PubMed Web of Science ®Google Scholar
• HAWLEY-NELSON P, CICCARONE V, GEBEYEHU G, JESSEJ, FELGNER PL: Lipofectamine reagent: a new higher ef-ficiency polycationic liposome transfection reagent. Focus (1993) 15:73–79.
   Google Scholar
• LEE ER, MARSHALL J, SIEGEL CS et al.: Detailed analysis ofstructures and formulations of cationic lipids for effi-cient gene transfer to the lung. Hum. Gene Ther. (1996) 7:1701–1717.
   PubMed Web of Science ®Google Scholar
• FELGNER JH, KUMAR R, SRIDHAR CN et al.: Enhancedgene delivery and mechanism studies with a novel se-ries of cationic lipid formulations. J. Biol. Chem. (1994) 269:2550–2561.
   PubMed Web of Science ®Google Scholar
• SAN H, YANG ZY, POMPILI VJ et al.: Safety and short-term toxicity of a novel cationic lipid formulation for human gene therapy. Hum. Gene Ther. (1993) 4:781–788.
   PubMed Web of Science ®Google Scholar
• WHEELER CJ, FELGNER PL, TSAI JT et al.: A novel cationiclipid greatly enhances plasmid DNA delivery and ex-pression in mouse lung. Proc. Nati Acad Sci. USA (1996) 93:11454–11459.
   PubMed Web of Science ®Google Scholar
• CAPLEN NJ, ALTON, E, MIDDLETON PG et al: Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Merl. (1995) 1:35–46.
   Google Scholar
• GILL DR, SOUTHERN KW, MOFFORD KA et al.: A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. (1997) 4:199–209.
   PubMed Web of Science ®Google Scholar
• LASIC DD: Recent developments in medical applica-tions of liposomes: sterically stabilized liposomes in cancer therapy and gene delivery in vivo. J. Contr. Rel. (1997) 48:203–222.
   Web of Science ®Google Scholar
• LEE ER, MARSHALL J, SIEGEL CS et al.: Detailed analysis ofstructures and formulations of cationic lipids for effi-cient gene transfer to the lung. Hum. Gene Ther. (1996) 7:1701–1717.
   PubMed Web of Science ®Google Scholar
• EASTMAN SJ, SIEGEL C, TOUSIGNANT J, SMITH AE,CHENG SH, SCHEULE RK: Biophysical characterisation of cationic lipid:DNA complexes. Biochim. Biophis. Acta (1997) 1325:41-62. 24BYK G, DUBERTRET C, ESCRIOU V etal. Synthesis, activity and structure-activity relationship studies of novel cationic lipids for DNA transfer. J. Merl. Chem. (1998) 41(2)224–235.
   Google Scholar
• BYK G, FREDERIC M, SCHERMAN D: One pot synthesis of unsymmetrically functionalised polyamines by a solid phase strategy starting from their symmetrical polyamine-counterparts. Tetrahedron Lett. (1997) 38:3219–3222.
   Google Scholar
• ESCRIOU V, CIOLINA C, LACROIX F, BYK G, SCHERMAND, WILS P: Cationic lipid-mediated gene transfer: effect of serum on cellular uptake and intracellular fate of lipopolyamine/DNA complexes. Biochim. Biophys. Acta (1998) 1368:276–288.
   PubMed Web of Science ®Google Scholar
• PITARD B, AGUERRE O, ARIAU M et al: Virus-sized selfassembling lamellar complexes between DNA and cationic micelles promote gene transfer. Proc. Natl. Acad. ScL USA (1997) 94:14412–14417.
   PubMed Web of Science ®Google Scholar
• WALKER S, SOFIA M, KAKARLA R et al.: Cationic facialamphiphiles: a promising class of transfection agents. Proc. Natl. Acad. ScL USA (1996) 93:1585–1590.
   PubMed Web of Science ®Google Scholar
• HAWLEY-NELSON P, CICCARONE V, GEBEYEHU G, JESSEJ, FELGNER PL: Lipofectamine reagent: a new higher ef-ficiency polycationic liposome transfection reagent. Focus (1993) 15:73–79.
   Google Scholar
• RUYSSCHAERT JM, EL OUAHABI A, WILLEAUME V et al.: Anovel cationic amphiphile for transfection of mam-malian cells. Biochem. Biophys. Res. Comm. (1994) 203:1622–1628.
   PubMed Web of Science ®Google Scholar
• EL OUAHABI A, PECTOR V, FUKS R, VANDENBRANDENM, RUYSSCHAERT JM: Double long-chain amidine liposome-mediated self replicating RNA transfection. Acta Clinica Belgica (1996) 51(3):194.
   Google Scholar
• BYK G, DUBERTRET C, SCHWARTZ B et al: Novel nonvi-ral vectors for gene delivery: synthesis and applica-tions. Letts. Peptide ScL (1997) 4:263–268.
   Google Scholar
• VIGNERON JP, OUDRHIRI N, FAUQUET M et al.: Guanidinium-cholesterol cationic lipids: efficient vec-tors for the transfection of eukaryotic cells. Proc. Natl. Acad. ScL USA (1996) 93:9682–9686.
   PubMed Web of Science ®Google Scholar
• OUDRHIRI N, VIGNERON JP, PLEUCHMAR M, LECLERC T,LEHN JM, LEHN P: Gene transfer by guanidinium-cholesterol cationic lipids into airway epithelial cells in vitro and in vivo. Proc. Natl. Acad. ScL USA (1996) 94:1651–1656.
   Google Scholar
• BYK G, SOTO J, MATTLER C, FREDERIC M, SCHERMAN D: Novel non-viral vectors for gene delivery: synthesis of a second-generation library of mono-functionalized (poly-guanidinium)amines and their introduction into cationic lipids. Biotech. Bioeng. Com. Chem. (1998) 2. (In Press).
   Google Scholar
• BEHR JP: Gene transfer with synthetic cationic amphi-philes: prospects for gene therapy. Bioconjugate Chem. (1994) 5:382–389.
   PubMed Web of Science ®Google Scholar
• STERNBERG B, SORGI F, HUANG L: New structures incomplex formation between DNA and cationic lipo-somes visualized by freeze-fracture electron micros-copy. FEBS Lett. (1994) 356:361–366.
   PubMed Web of Science ®Google Scholar
• KOLTOVER I, SALDITT T, RADLER JO, SAFINYA CR: An in-verted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery. Science (1998) 281:78–81.
   PubMed Web of Science ®Google Scholar
• LASIC DD, STREY H, STUART MCA, PODGORNIK R, FRE-DERIK PM: The structure of DNA-Liposome complexes. J. Am. Chem. Soc. (1997) 119:832–833.
   Web of Science ®Google Scholar
• RADLER JO, KOLTOVER I, SALDITT T, SAFINYA CR: Struc-ture of DNA-cationic liposome complexes: DNA inter-calation in multilamellar membranes in distinct interhelical packing regimes. Science (1997) 275:810.
   PubMed Web of Science ®Google Scholar
• ZUIDAM NJ, BARENHOLTZ Y: Electrostatic parameters of cationic liposomes commonly used for gene deliv-ery as determined by 4-heptadecy1-7-hydroxycoumarin. Biochim. Biophys. Acta (1997) 1329:211–222.
   PubMed Web of Science ®Google Scholar
• ZUIDAM NJ, BARENHOLTZ Y: Electrostatic and struc-tural properties of complexes involving plasmid DNA and cationic lipids commonly used for gene delivery. Biochim. Biophys. Acta (1998) 1368:115–128.
   PubMed Web of Science ®Google Scholar
• HIRSCH-LERNER D, BARENHOLTZ Y: Probing DNA-cationic lipid interactions with the fluorophore tri-methylammonium diphenyl-hexatriene (TMADPH). Biochim. Biophys. Acta (1998) 1370:17–30.
   PubMed Web of Science ®Google Scholar
• BOUKHNIKACHVILI T, AGUERRE-CHARIOL O, AIRIAU M, LESIEUR S, OLLIVON J, VACUS J: Structure of in-serum transfecting DNA-cationic lipid complexes. FEBS Lett. (1997) 409:188–194.
   PubMed Web of Science ®Google Scholar
• BLESSING T, RÉMY JS, BEHR JP: Molecular collapse of plasmid DNA into stable virus-like particles. Proc. Natl. Acad. ScL USA (1998) 95:1427–1431.
   PubMed Web of Science ®Google Scholar
• HARBOTTLE RP, COOPER RG, HART SL et al.: An RGD-oligolysine peptide: a prototype construct for integrin-mediated gene delivery. Hum. Gene Ther. (1998) 9:1037–1047.
   PubMed Web of Science ®Google Scholar