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Expert Opinion on Therapeutic Patents Volume 12, 2002 - Issue 3
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Miscellaneous

Antisense strategies and non-viral gene therapy for cancer

K Helen Bremner Cancer Research UK Institute for Cancer Studies, University of Birmingham, Vincent Drive, Birmingham B15 2TT, UK, Tel.: +44 121 414 3290; Fax: +44 121 414 3263; , E-mail: [email protected]
&
Martin L Read Cancer Research UK Institute for Cancer Studies, University of Birmingham, Vincent Drive, Birmingham B15 2TT, UK, Tel.: +44 121 414 3290; Fax: +44 121 414 3263; , E-mail: [email protected]
Pages 379-391 | Published online: 25 Feb 2005

Bibliography

  • HACKETT NR, KAMINSKY SM, D, CRYSTAL RG: Antivector and antitransgene host responses in gene therapy. Curr. Opin. Mol. Ther. (2000) 2:376–382.
  • CUNNINGHAM CC, HOLMLUND JT, GEARY RS et al.: A Phase I trial of H-ras antisense oligonucleotide ISIS 2503 administered as a continuous intravenous infusion in patients with advanced carcinoma. Cancer (2001) 92:1265–1271.
  • LIU F, SONG Y, LIU D: Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. (1999) 6:1258–1266.
  • ZHANG G, SONG YK, LIU D: Long-term expression of human al-antitrypsin gene in mouse liver achieved by intravenous administration of plasmid DNA using a hydrodynamics-based procedure. Gene Ther. (2000) 7:1344–1349.
  • PEDROSO DE LIMA MC, SIMOES S, PIRES P, FANECA H, DUZGUNES N: lipid-DNA complexes in gene delivery: from biophysics to biological applications. Adv. Drug Deliv. Rev (2001) 47:277–294.
  • WAGNER E, OGRIS M, ZAUNER W: Polylysine-based transfection systems utilizing receptor-mediated delivery. Adv. Drug Deliv. Rev (1998) 30:97–113.
  • BOUSSIF O, LEZOUALCH F, ZANTA MA et al.: A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. USA (1995) 92:7297–7301.
  • GODBEY WT, WU KK, MIKOS AG: Poly(ethylenimine) and its role in gene delivery.' Control. Release (1999) 60:149–160.
  • SCHATZLEIN AG: Non-viral vectors in gene therapy: principles and progress. Anticancer Drugs (2001) 12:275–304.
  • •Comprehensive review of non-viral vectors in cancer gene therapy.
  • KIRCHEIS R, BLESSING T, BRUNNER S, WIGHTMAN L, WAGNER E: Tumor targeting with surface-shielded ligand-polycation DNA complexes. " Control. Release (2001) 72:165–170.
  • YOO GH, HUNG MC, LOPEZ- G et al.: Phase I trial of intratumoral liposome ElA gene therapy in patients with recurrent breast and head and neck cancer. Clin. Cancer Res. (2001) 7:1237–1245.
  • GLEICH LL, GLUCKMAN JL, NEMUNAITIS J et al.: Clinical experience with HLA-I37 plasmid DNA/lipid complex in advanced squamous cell carcinoma of the head and neck. Arch. Otolaryngol. Head Neck Surg. (2001) 127:775–779.
  • ••A clinical study showing that intraturnoralinjections of Allovectin-7, a DNA/lipid complex designed to express the class I major histocompatibility complex antigen HLA-B7, in patients with advanced head and neck squarnous cell carcinoma is safe and can produce responses in overall disease.
  • STOPECK AT, JONES A, HERSH EM et al.: Phase II study of direct intralesional gene transfer of allovectin-7, an HLA-I37/132-microglobulin DNA-liposome complex in patients with metastatic melanoma. Clin. Cancer Res. (2001) 7:2285–2291.
  • ••A Phase II study showing that intraturnoraladministration of Allovectin-7 in metastatic melanoma is safe and can produce both responses in injected lesions and in overall
  • HORTOBAGYI GN, UENO NT, XIA W et al.: Cationic liposome-mediated ElA gene transfer to human breast and ovarian cancer cells and its biologic effects: a Phase I clinical trial. J. Clin. Oncol. (2001) 19:3422–3433.
  • ••A Phase I trial showing the feasibility ofintracavitary injection of the ElA gene complexed with cationic liposomes in patients with both HER-2/neu-overexpressing and low HER-2/neu-expressing breast and ovarian cancers.
  • CHEN QR, KUMAR D, STASS SA, MIXSON AJ: Liposomes complexed to plasmids encoding angiostatin and endostatin inhibit breast cancer in nude mice. Cancer Res. (1999) 59:3308–3312.
  • ZOU Y, ZONG G, LING YH et al: Effective treatment of early endobronchial cancer with regional administration of liposome-p53 complexes." Natl. Cancer Inst. (1998) 90:1130–1137.
  • ZOU Y, ZONG G, LING YH, PEREZ-SOLER R: Development of cationic liposome formulations for intratracheal gene therapy of early lung cancer. Cancer Gene The]: (2000) 7:683–696.
  • RAMESH R, SAEKI T, TEMPLETON NS et al.: Successful treatment of primary and disseminated human lung cancers by systemic delivery of tumor suppressor genes using an improved liposome vector. Mol. Ther. (2001) 3:337–350.
  • •This paper describes the use of improved extruded DOTAP:cholesterol cationic liposomes for the delivery of tumour suppressor genes to localised human primary lung cancers and to experimental disseminated metastases.
  • XU L, PIROLLO KF, TANG WH, RAIT A, CHANG EH: Transferrin-liposome-mediated systemic p53 gene therapy in combination with radiation results in regression of human head and neck cancer xenografts. Hum. Gene Ther. (1999) 10:2941–2952.
  • •This study demonstrates tumour-specific gene delivery of the p..53gene by iv. administration of transferrin-targeted lipoplexes resulting in sensitisation of mouse xenograft tumours to radiotherapy.
  • YANG JP, HUANG L: Time-dependent maturation of cationic liposome-DNA complex for serum resistance. Gene Ther. (1998) 5:380–387.
  • YANG JP, HUANG L: Overcoming the inhibitory effect of serum on lipofection by the charge ratio of cationic liposome to DNA. Gene Ther. (1997) 4:950–960.
  • WHEELER JJ, PALMER L, OSSANLOU et al.: Stabilized plasmid-lipid particles: construction and characterization. Gene The]: (1999) 6:271–281.
  • XU L, PIROLLO KF, CHANG EH: Tumor-targeted p53-gene therapy enhances the efficacy of conventional chemo/ radiotherapy. J. Control. Release (2001) 74:115–128.
  • ZHANG YP, SEKIROV L, SARAVOLAC EG et al.: Stabilized plasmid-lipid particles for regional gene therapy: formulation and transfection properties. Gene Ther. (1999) 6:1438–1447.
  • TAMP, MONCK M, LEE D et al.: plasmid-lipid particles for systemic gene therapy. Gene Ther. (2000) 7:1867–1874.
  • •One of the first studies to show prolonged blood circulation of stabilised lipoplexes in mice and significant accumulation at a distal tumour site. The levels of gene expression at the tumour site were modest.
  • COLL JL, CHOLLET P, BRAMBILLA E, DESPLANQUES D, BEHR JP, FAVROT: In vivo delivery to tumors of DNA complexed with linear polyethylenimine. Hum. Gene The]: (1999) 10:1659–1666.
  • AOKI K, FURUHATA S, HATANAKA K et al.: Polyethylenimine-mediated gene transfer into pancreatic tumor dissemination in the murine peritoneal cavity. Gene Ther. (2001) 8:508–514.
  • GAUTAM A, DENSMORE CL, WALDREP JC: Inhibition of experimental lung metastasis by aerosol delivery of PEI-p53 complexes. Mol. Ther. (2000) 2:318–323.
  • •This study shows that delivery of the p.53 gene by aerosol using PEI can inhibit the growth of lung metastasis.
  • DASH PR, READ ML, BARRETT LB, WOLFERT MA, SEYMOUR LW: Factors affecting blood clearance and in vivo distribution of polyelectrolyte complexes for gene delivery. Gene The]: (1999) 6:643–650.
  • WARD CM, READ ML, SEYMOUR LW: Systemic circulation of poly(L-lysine)/DNA vectors is influenced by polycation molecular weight and type of DNA: differential circulation in mice and rats and the implications for human gene therapy. Blood (2001) 97:2221–2229.
  • DASH PR, READ ML, FISHER KD et al.: binding to proteins and cells of polymeric gene delivery vectors surface modified with a multivalent hydrophilic polymer and retargeting through attachment of transferrin. J. Biol. Chem. (2000) 275:3793–3802.
  • FISHER KD, ULBRICH K, SUBR V et al: A versatile system for receptor-mediated gene delivery permits increased entry of DNA into target cells, enhanced delivery to the nucleus and elevated rates of transgene expression. Gene Ther. (2000) 7:1337–1343.
  • OGRIS M, BRUNNER S, SCHULLER S, KIRCHEIS R, WAGNER E: PEGylated DNA/transferrin-PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery. Gene Ther. (1999) 6:595–605.
  • KIRCHEIS R, WIGHTMAN L, SCHREIBER A et al.: Polyethylenimine/ DNA complexes shielded by transferrin target gene expression to tumors after systemic application. Gene Ther. (2001) 8:28–40.
  • •Demonstration of preferential tumour-targeting after systemic application of transferrin-shielded PEI/DNA polyplexes into mice bearing subcutaneously growing Neuro2a tumours.
  • NEMUNAITIS J, HOLMLUND JT, KRAYNAK M et al.: Phase I evaluation of ISIS 3521, an antisense oligodeoxynucleotide to protein kinase C-a, in patients with advanced cancer. j Clin. Oncol. (1999) 17:3586–3595.
  • ••A Phase I trial showing clinical activity an antisense oligonucleotide to PKCa.
  • YUEN AR, HALSEY J, FISHER GA et al.:Phase I study of an antisense oligonucleotide to protein kinase C-a (ISIS 3521/CGP 64128A) in patients with cancer. Clin. Cancer Res. (1999) 5:3357–3363.
  • STEVENSON JP, YAO KS, GALLAGHERM et al.: Phase I clinical/pharmacokinetic and pharmacodynamic trial of the c-raf-1 antisense oligonucleotide ISIS 5132 (CGP 69846A). J. Clin. Oncol. (1999) 17:2227–2236.
  • CUNNINGHAM CC, HOLMLUND JT, SCHILLER JH et al: A Phase I trial of c-Raf kinase antisense oligonucleotide ISIS 5132 administered as a continuous intravenous infusion in patients with advanced cancer. Clin. Cancer Res. (2000) 6:1626–1631.
  • WATERS JS, WEBB A, CUNNINGHAM et al.: Phase I clinical and study of bc1-2 antisense oligonucleotide therapy in patients with non-Hodgkin's lymphoma. j. Clin. Oncol. (2000) 18:1812–1823.
  • JANSEN B, WACHECK V, HEERE-RESS et al.: Chemosensitisation of malignant by BCL2 antisense therapy. Lancet (2000) 356: 1728-1733.
  • ••A Phase I-II clinical study investigating thecombination of bc/-2antisense therapy and dacarbazine.
  • HOLMLUND JT, MONIA BP, KWOH TJ, DORR FA: Toward antisense oligonucleotide therapy for cancer: ISIS compounds in clinical development. Can: Opin. Mol. Ther. (1999) 1:372–385.
  • ••Review summarising the status ofdevelopment of three anticancer antisense oligonucleotides from Isis Pharmaceuticals.
  • PARK YG, NESTEROVA M, AGRAWAL S, CHO-CHUNG YS: Dual blockade of cyclic AMP response element- (CRE) and AP-1-directed transcription by CRE-transcription factor decoy oligonucleotide. gene-specific inhibition of tumor growth. J.. Chem. (1999) 274:1573–1580.
  • ALPER O, BERGMANN-LEITNER ES, ABRAMS S, CHO-CHUNG YS:, growth arrest and suppression of invasiveness by CRE-decoy oligonucleotide in ovarian cancer cells: protein kinase A downregulation and cytoplasmic export of CRE-binding proteins. Mol. Cell. Biochem. (2001) 218:55–63.
  • BENIMETSKAYA L, TAKLE GB, VILENCHIK M, LEBEDEVA I, MILLER P, STEIN CA: Cationic porphyrins: novel delivery vehicles for antisense oligodeoxynucleotides. Nucleic Acids Res. (1998) 26:5310–5317.
  • ZANGEMEISTER-WITTKE U, LEECH SH, OLIE RA et al.: A novel bispecific antisense oligonucleotide inhibiting both bc1-2 and bc1-xL expression efficiently induces apoptosis in tumor cells. Clin. Cancer Res. (2000) 6:2547–2555.
  • ••The first report of a bc/-2/bc/-xL bispecificantisense oligonucleotide that can induce apoptotic cell death in small cell and non-small cell lung cancer cell lines.
  • STRASBERG RIEBER M,-WITTKE U, RIEBER: p53-Independent induction of apoptosis in human melanoma cells by a bc1-2/bc1-xL bispecific antisense oligonucleotide. Res. (2001) 7:1446–1451.
  • GAUTSCHI O, TSCHOPP S, OLIE RA etal.: Activity of a novel bc1-2/bc1-xL-bispecific antisense oligonucleotide against tumors of diverse histologic origins.' Nati Cancer Inst. (2001) 93:463–471.
  • KLASA RJ, BALLY MB, NG R, GOLDIE JH, GASCOYNE RD, WONG FM: of human non-Hodgkin's lymphoma in SCID mice by BCL-2 antisense oligonucleotides combined with low-dose cyclophosphamide. Clin. Cancer Res. (2000) 6:2492–2500.
  • CHEN L, AGRAWAL S, ZHOU W, ZHANG R, CHEN J: Synergistic activation of p53 by inhibition of MDM2 expression and DNA damage. Proc. Nati Acad. Sci. USA (1998) 95:195–200.
  • MAHATO RI, TAKEMURA S, AKAMATSU K, NISHIKAWA M, TAKAKURA Y, HASHIDA M: and disposition of antisense oligonucleotides complexed with glycosylated poly(L-lysine).. Pharmacol. (1997) 53:887–895.
  • VINOGRADOV SV, BRONICH TK, KABANOV AV: Self-assembly of polyamine-poly(ethylene glycol) copolymers with phosphorothioate oligonucleotides.. Chem. (1998) 9:805–812.
  • KABANOV AV, VINOGRADOV SV, SUZDALTSEVA YG, ALAKHOV V:-soluble block polycations as carriers oligonucleotide delivery. Bioconjug. Chem. (1995) 6:639–643.
  • READ ML, DASH PR, CLARK A et al.:Physicochemical and biological characterisation of an antisense oligonucleotide targeted against the bc1-2 mRNA complexed with cationic-hydrophilic copolymers. Ear: I Pharm. Sci. (2000) 10:169–177.
  • HARADA A, TOGAWA H, KATAOKA K: Physicochemical properties and nuclease resistance of antisense-oligodeoxynucleotides entrapped in the core of polyion complex micelles composed of poly(ethylene glycol)-poly(L-lysine) block copolymers. Eur.J Pharm. Sci. (2001) 13:35–42.
  • MUNDIGL O, OCHOA GC, DAVID C, SLEPNEV VI, KABANOV A, DE CAMILLI P: Amphiphysin I antisense oligonucleotides inhibit neurite outgrowth in cultured hippocampal neurons.. (1998) 18:93–103.
  • ROY S, ZHANG K, ROTH T, S, KAO RS, KABANOV A: Reduction of fibronectin expression by intravitreal administration of antisense oligonucleotides. Nat. Biotechnol. (1999) 17:476–479.
  • YU RZ, GEARY RS, LEEDS JM et al.: and tissue disposition in monkeys of an antisense oligonucleotide inhibitor of Ha-ras encapsulated in stealth liposomes. Pharm. Res. (1999) 16:1309–1315.
  • •This study shows that stealth liposomes can protect antisense oligonucleotides from nucleases and slow the rate of clearance from the systemic circulation following iv. administration to rhesus monkeys.
  • TEMPLIN MV, LEVIN AA, GRAHAM MJ et al.: Pharmacokinetic and toxicity profile of a phosphorothioate oligonucleotide following inhalation delivery to lung in mice. Antisense Nucleic Acid Drug. Dev. (2000) 10:359–368.
  • BETTINGER T, READ ML: Recent developments in RNA-based strategies for cancer gene therapy. Can: Opin. Mol. Ther. (2001) 3:116–124.
  • MCGUFFIE EM, PACHECO D, CARBONE GM, CATAPANO CV: Antigene and antiproliferative effects of a c-myc-targeting phosphorothioate triple helix-forming oligonucleotide in human leukemia cells. Cancer Res. (2000) 60:3790–3799.
  • MOLOGNI L, MARCHESI E, NIELSEN PE, GAMBACORTI-PASSERINI C: of promyelocytic leukemia (PML)/retinoic acid receptor-a and PML expression in acute promyelocytic leukemia cells by anti-PML peptide nucleic acid. Res. (2001) 61:5468–5473.
  • PIVA R, DEL SENNO L, LAMBERTINI E, PENOLAZZI L, NASTRUZZI C: of estrogen receptor gene transcription in breast cancer cells by liposome delivered decoy molecules. Biochem. Mol. Biol. (2000) 75:121–128.
  • MICKLEFIELD J: Backbone modificationof nucleic acids: synthesis, structure and therapeutic applications. Can: Med. Chem. (2001) 8:1157–1179.
  • GRYAZNOV SM: Oligonucleotide N3'-P5' phosphoramidates as potential therapeutic agents. Biochlin. Biophys. Acta. (1999) 1489:131–140.
  • LYER RP, ROLAND A, ZHOU W, GHOSH K: Modified oligonucleotides-
  • ••, properties and applications. Carr. Opin. MM. Ther. (1999) 1:344–358.
  • HERDEWIJN P: Heterocyclic of oligonucleotides and antisense technology. Antisense Nucleic Acid Drug Dev. (2000) 10:297–310.
  • SIOUD M, SORENSEN DR: A nuclease-resistant protein kinase C a ribozyme blocks glioma cell growth. Nat. Biotechnol (1998) 16:556–561.
  • •This important study shows that a nuclease-resistant ribozyme can inhibit the proliferation of glioma tumor cells in inbred syngeneic rats.
  • PAVCO PA, BOUHANA KS, GALLEGOS AM et al.: Antitumor and antimetastatic activity of ribozymes targeting the messenger RNA of vascular endothelial growth factor receptors. Gin. Cancer Res. (2000) 6:2094–2103.
  • ••First demonstration that syntheticribozymes targeting vascular endothelial growth factor receptor mRNA can reduce the growth and metastasis of solid tumors in vivo.
  • SANDBERG JA, BOUHANA KS, GALLEGOS AM et al.: Pharmacokinetics of an antiangiogenic ribozyme (ANGIOZYME) in the mouse. Antisense Nucleic Acid Drug. Dev. (1999) 9:271–277.
  • SANDBERG JA, SPROUL CD, BLANCHARD KS et al.: Acute toxicology and pharmacokinetic assessment of a ribozyme (ANGIOZYME) targeting vascular endothelial growth factor receptor mRNA in the cynomolgus monkey. Nucleic Acid Drug. Dev. (2000) 10:153–162.
  • •Evaluation of anti-Flt-1 ribozyme in cynomolgus monkeys following iv. infusion or Sc. injection. The ribozyme was well tolerated with no clear evidence of adverse effects. There were slight increases in spleen weight and lymphoid hyperplasia in several animals but these changes were not dose dependent.
  • HENRY SP, GICLAS PC, LEEDS J et al:Activation of the alternative pathway of complement by a phosphorothioate oligonucleotide: potential mechanism of action. Pharmacol. Exp. Ther. (1997) 281:810–816.
  • GALBRAITH WM, HOBSON WC, GICLAS PC, SCHECHTER PJ, AGRAWAL S: Complement activation and hemodynamic changes following intravenous administration of phosphorothioate oligonucleotides in the. Antisense Res. Dev. (1994) 4:201–206.
  • OUPICKY D, CARLISLE RC, SEYMOUR LW: Triggered intracellular activation of disulfide crosslinked polyelectrolyte gene delivery complexes with extended systemic circulation in vivo. Gene Ther. (2001) 8:713–724.
  • BALAKIREV M, SCHOEHN G, CHROBOCZEK J: Lipoic acid-derived amphiphiles for redox-controlled DNA delivery. Chem. Biol. (2000) 7:813–819.
  • OUPICKY D, PARKER AL, SEYMOUR LW: Laterially stabilized complexes of DNA with linear reducible polycations: strategy for triggered intracellular activation of DNA delivery vectors. Am. Chem. Soc. (2002) 124:8–9.
  • SIMOES S, SLEPUSHKIN V, PIRES P, GASPER R, PEDROSO DE LIMA MC, DUZGUNES N: Human serum albumin enhances DNA transfection by lipoplexes and confers resistance to inhibition by serum. Biochim. Biophys. Acta. (2000) 1463:459–469.
  • BREMNER KH, SEYMOUR LW, POUTON CW: Harnessing nuclear localization pathways for transgene delivery. Carr. Opin.Mol. Ther. (2001) 3:170–177.
  • BRANDEN LJ, MOHAMED AJ, SMITH CI: A peptide nucleic acid-nuclear localization signal fusion that mediates nuclear transport of DNA. Nat. Biotech. (1999) 17:784–787.
  • ZANTA MA, BELGUISE-VALLADIER P,BEHR JP: Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc. Nati Acad. Sci. USA (1999) 96:91–96.
  • •Linked a single NLS-peptide to one end of a gene and showed a 10- to 1000-fold increase in reporter gene activity.
  • LI X, STUCKERT P, BOSCH I, MARKS JD, MARASCO WA: Single-chain antibody-mediated gene delivery into ErbB2-positive human breast cancer cells. Cancer Gene Ther. (2001) 8:555–565.
  • SPEAR MA, BREAKEFIELD XO, BELTZER J et al: Isolation, characterization, and recovery of small peptide phage display epitopes selected against viable malignant glioma cells. Cancer Gene Ther. (2001) 8:506–511.
  • SU Z, LEBEDEVA IV, GOPALKRISHNAN RV et al: A approach for selectively inducing programmed cell death in human cancer cells. Proc. Natl. Acad. Sci. USA (2001) 98:10332–10337.
  • ••An intriguing approach consisting of acancer-specific apoptosis-inducing gene and an oncogene inactivation strategy that may provide the foundation for developing an effective therapy for pancreatic cancer.
  • RUDIN CM, HOLMLUND J, FLEMING GF et al.: Phase I trial of ISIS 5132, an antisense oligonucleotide inhibitor of c-raf-1, administered by 24-hour weekly infusion to patients with advanced cancer. Gin. Cancer Res. (2001) 7:1214–1220.
  • CHEN HX, MARSHALL JL, NESS E et al.: A safety and pharmacokinetic study of a mixed-backbone oligonucleotide (GEM231) targeting the Type I protein kinase A by two-hour infusions in patients with refractory solid tumors. Gin. Cancer Res. (2000) 6:1259–1266.

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