Gene Therapy of the Typical Multidrug Resistance Phenotype of Cancers: A New Hope?

Stefkova, 2004, ATP-binding cassette (ABC) transporters in human metabolism and diseases, Physiol Res, 53, 235, 10.33549/physiolres.930432 Scheffer, 1995, The drug resistance-related protein LRP is the human major vault protein, Nat Med, 1, 578, 10.1038/nm0695-578 Rappa, 1999, New insights into the biology and pharmacology of the multidrug resistance protein (MRP) from gene knockout models, Biochem Pharmacol, 58, 557 Vasanthakumar, 1989, Modulation of drug resistance in a daunorubicin resistant subline with oligonucleoside methylphosphonates, Cancer Commun, 1, 225 Alahari, 1996, Inhibition of expression of the multidrug resistance-associated P-glycoprotein of by phosphorothioate and 5′ cholesterol-conjugated phosphorothioate antisense oligonucleotides, Mol Pharmacol, 50, 808 Cucco, 1996, In vitro and in vivo reversal of multidrug resistance in a human leukemia-resistant cell line by mdr1 antisense oligodeoxynucleotides, Cancer Res, 56, 4332 Motomura, 1998, Inhibition of P-glycoprotein and recovery of drug sensitivity of human acute leukemic blast cells by multidrug resistance gene (mdr1) antisense oligonucleotides, Blood, 91, 3163, 10.1182/blood.V91.9.3163 Dassow, 1998, Modulation of P-glycoprotein-mediated multidrug resistance in a doxorubicin-resistant subline of the human lymphoblastoid cell line CCRF-CEM by phosphorothioate antisense oligonucleotides, Int J Clin Pharmacol Ther, 36, 93 Yaswen, 1993, Effects of sequence of thioated oligonucleotides on cultured human mammary epithelial cells, Antisense Res Dev, 3, 67, 10.1089/ard.1993.3.67 Vaerman, 1995, BCR-ABL antisense oligodeoxyribonucleotides suppress the growth of leukemic and normal hematopoietic cells by a sequence-specific but nonantisense mechanism, Blood, 86, 3891, 10.1182/blood.V86.10.3891.bloodjournal86103891 Crooke, 1992, Therapeutic applications of oligonucleotides, Annu Rev Pharmacol Toxicol, 32, 329, 10.1146/annurev.pa.32.040192.001553 Brigui, 2003, Minimally modified phosphodiester antisense oligodeoxyribonucleotide directed against the multidrug resistance gene mdr1, Biochem Pharmacol, 65, 747, 10.1016/S0006-2952(02)01558-7 Kruger, 1982, Self-splicing RNA, Cell, 31, 147, 10.1016/0092-8674(82)90414-7 Guerrier-Takada, 1983, The RNA moiety of ribonuclease P is the catalytic subunit of the enzyme, Cell, 35, 849, 10.1016/0092-8674(83)90117-4 Uhlenbeck, 1987, A small catalytic oligoribonucleotide, Nature, 328, 596, 10.1038/328596a0 Buzayan, 1986, Nucleotide sequence and newly formed phosphodiester bond of spontaneously ligated satellite tobacco ringspot virus RNA, Nucleic Acids Res, 14, 9729, 10.1093/nar/14.24.9729 Kobayashi, 2001, Novel approaches to reversing anti-cancer drug resistance using gene-specific therapeutics, Hum Cell, 14, 172 Gao, 1999, Tumor-specific expression of anti-mdr1 ribozyme selectively restores chemosensitivity in multidrug-resistant colon-adenocarcinoma cells, Int J Cancer, 82, 346, 10.1002/(SICI)1097-0215(19990730)82:3<346::AID-IJC7>3.0.CO;2-# Palfner, 1995, Improvement of hammerhead ribozymes cleaving mdr-1 mRNA, Biol Chem Hoppe Seyler, 376, 289, 10.1515/bchm3.1995.376.5.289 Kiehntopf, 1994, Ribozyme-mediated cleavage of the MDR-1 transcript restores chemosensitivity in previously resistant cancer cells, EMBO J, 13, 4645, 10.1002/j.1460-2075.1994.tb06787.x Holm, 1995, Similar cleavage efficiencies of an oligoribonucleotide substrate and an mdr1 mRNA segment by a hammerhead ribozyme, Gene, 167, 221, 10.1016/0378-1119(95)00709-1 Morassutti, 1999, Reduction of mdr1 gene amplification in human multidrug-resistant LoVo DX cell line is promoted by triple helix-forming oligonucleotides, Antisense Nucleic Acid Drug Dev, 9, 261, 10.1089/oli.1.1999.9.261 Sedelnikova, 2000, Targeting the human mdr1 gene by 125I-labeled triplex-forming oligonucleotides, Antisense Nucleic Acid Drug Dev, 10, 443, 10.1089/oli.1.2000.10.443 Marthinet, 2000, Modulation of the typical multidrug resistance phenotype by targeting the MED-1 region of human MDR1 promoter, Gene Ther, 7, 1224, 10.1038/sj.gt.3301231 Labialle, 2004, New invMED1 element cis-activates human multidrug-related MDR1 and MVP genes, involving the LRP130 protein, Nucleic Acids Res, 32, 3864, 10.1093/nar/gkh722 McManus, 2002, Gene silencing in mammals by small interfering RNAs, Nat Rev Genet, 3, 737, 10.1038/nrg908 Dillin, 2003, The specifics of small interfering RNA specificity, Proc Natl Acad Sci U S A, 100, 6289, 10.1073/pnas.1232238100 Tuschl, 2002, Expanding small RNA interference, Nat Biotechnol, 20, 446, 10.1038/nbt0502-446 Nieth, 2003, Modulation of the classical multidrug resistance (MDR) phenotype by RNA interference (RNAi), FEBS Lett, 545, 144, 10.1016/S0014-5793(03)00523-4 Wu, 2003, Small interfering RNA-induced suppression of MDR1 (P-glycoprotein) restores sensitivity to multidrug-resistant cancer cells, Cancer Res, 63, 1515 Yague, 2004, Complete reversal of multidrug resistance by stable expression of small interfering RNAs targeting MDR1, Gene Ther, 11, 1170, 10.1038/sj.gt.3302269 Xu, 2004, Strategies for inhibition of MDR1 gene expression, Mol Pharmacol, 66, 268, 10.1124/mol.66.2.268 Hua, 2005, Stable suppression of MDR-1 gene using siRNA expression vector to reverse drug resistance in a human uterine sarcoma cell line, Gynecol Oncol, 98, 31, 10.1016/j.ygyno.2005.03.042 Zhang, 2005, Reversal of multidrug resistance by small interfering double-stranded RNAs in ovarian cancer cells, Gynecol Oncol, 97, 501, 10.1016/j.ygyno.2005.01.027 Bartsevich, 2000, Regulation of the MDR1 gene by transcriptional repressors selected using peptide combinatorial libraries, Mol Pharmacol, 58, 1, 10.1124/mol.58.1.1 Xu, 2002, Selective inhibition of P-glycoprotein expression in multidrug-resistant tumor cells by a designed transcriptional regulator, J Pharmacol Exp Ther, 302, 963, 10.1124/jpet.102.033639 Kuznetsova, 2003, Catalytic DNA and RNA for targeting MDR1 mRNA, Nucleosides Nucleotides Nucleic Acids, 22, 1521, 10.1081/NCN-120023025 Niewiarowski, 2000, Multidrug resistance-associated protein–reduction of expression in human leukaemia cells by antisense phosphorothioate olignucleotides, Acta Biochim Pol, 47, 1183, 10.18388/abp.2000_3971 Peaston, 2001, MRP1 gene expression level regulates the death and differentiation response of neuroblastoma cells, Br J Cancer, 85, 1564, 10.1054/bjoc.2001.2144 Kawabata, 2001, Breast cancer resistance protein directly confers SN-38 resistance of lung cancer cells, Biochem Biophys Res Commun, 280, 1216, 10.1006/bbrc.2001.4267 Hatanaka, 2001, Modulation of multidrug resistance in a cancer cell line by anti-multidrug resistance-associated protein (MRP) ribozyme, Anticancer Res, 21, 879 Nagata, 2002, Reversal of drug resistance using hammerhead ribozymes against multidrug resistance-associated protein and multidrug resistance 1 gene, Int J Oncol, 21, 1021 Kowalski, 2002, Modulation of the atypical multidrug-resistant phenotype by a hammerhead ribozyme directed against the ABC transporter BCRP/MXR/ABCG2, Cancer Gene Ther, 9, 579, 10.1038/sj.cgt.7700471 Akiyama, 1999, Mechanisms for resistance to anticancer agents and the reversal of the resistance, Hum Cell, 12, 95 Kobayashi, 1994, Reversal of drug sensitivity in multidrug-resistant tumor cells by an MDR1 (PGY1) ribozyme, Cancer Res, 54, 1271 Shi, 2004, Effective intracellular delivery of oligonucleotides in order to make sense of antisense, J Control Release, 97, 189, 10.1016/j.jconrel.2004.03.016 Roth, 2004, Engineering synthetic vectors for improved DNA delivery, Annu Rev Biomed Eng, 6, 397, 10.1146/annurev.bioeng.6.040803.140203 Desjardins, 1995, Inhibition of the rat cytochrome P450 3A2 by an antisense phosphorothioate oligodeoxynucleotide in vivo, J Pharmacol Exp Ther, 275, 1608 2000, Datamonitor. Antisense therapy: technical aspects and commercial opportunities, Reuters Business Insight, 187 Geary, 1997, Antisense oligonucleotide inhibitors for the treatment of cancer: 1. Pharmacokinetic properties of phosphorothioate oligodeoxynucleotides, Anticancer Drug Des, 12, 383 Astriab-Fisher, 2000, Antisense inhibition of P-glycoprotein expression using peptide-oligonucleotide conjugates, Biochem Pharmacol, 60, 83, 10.1016/S0006-2952(00)00310-5 Farzaneh, 1998, Gene therapy of cancer, Immunol Today, 19, 294, 10.1016/S0167-5699(98)01280-8 Deisseroth, 1999, Clinical trials involving multidrug resistance transcription units in retroviral vectors, Clin Cancer Res, 5, 1607 Eid, 1996, Multidrug resistance of testis cancers, Anticancer Res, 16, 3447 Ling, 1997, Multidrug resistance, Cancer Chemother Pharmacol, 40, S3, 10.1007/s002800051053 Galimberti, 2003, The clinical relevance of the expression of several multidrug-resistant-related genes in patients with primary acute myeloid leukemia, J Chemother, 15, 374, 10.1179/joc.2003.15.4.374 Schmitt, 2000, Genetic analysis of chemoresistance in primary murine lymphomas, Nat Med, 6, 1029, 10.1038/79542 Garraway, 2002, MDR1 inhibition, Eur J Cancer, 38, 2337, 10.1016/S0959-8049(02)00490-2 Cabral, 2001, Factors determining cellular mechanisms of resistance to antimitotic drugs, Drug Resist Update, 4, 3, 10.1054/drup.2000.0172 Schinkel, 1994, Disruption of the mouse mdr1a P-glycoprotein gene leads to a deficiency in the blood-brain barrier and to increased sensitivity to drugs, Cell, 77, 491, 10.1016/0092-8674(94)90212-7 Hesdorffer, 1998, Phase I trial of retroviral-mediated transfer of the human MDR1 gene as marrow chemoprotection in patients undergoing high-dose chemotherapy and autologous stem-cell transplantation, J Clin Oncol, 16, 165, 10.1200/JCO.1998.16.1.165 Eckert, 2000, Clinical scale production of an improved retroviral vector expressing the human multidrug resistance 1 gene (MDR1), Bone Marrow Transplant, 25, S114, 10.1038/sj.bmt.1702368 Persidis, 2000, Cancer multidrug resistance, Nat Biotechnol, 18, IT18, 10.1038/80051 Persidis, 1999, Cancer multidrug resistance, Nat Biotechnol, 17, 94, 10.1038/5289 Brown, 1998, The unique physiology of solid tumors, Cancer Res, 58, 1408 Kuriyama, 1997, Tissue-specific expression of HSV-tk gene can induce efficient antitumor effect and protective immunity to wild-type hepatocellular carcinoma, Int J Cancer, 71, 470, 10.1002/(SICI)1097-0215(19970502)71:3<470::AID-IJC27>3.0.CO;2-I Yan, 2001, Regulatory elements governing transcription in specialized myofiber subtypes, J Biol Chem, 276, 17361, 10.1074/jbc.M101251200 Latham, 2000, Prostate-specific antigen promoter/enhancer driven gene therapy for prostate cancer, Cancer Res, 60, 334 Swift, 1989, Differential requirements for cell-specific elastase I enhancer domains in transfected cells and transgenic mice, Genes Dev, 3, 687, 10.1101/gad.3.5.687 Anson, 1994, Transcriptional activity of the CD45 gene promoter in retroviral vector constructs, Biochim Biophys Acta, 1219, 81, 10.1016/0167-4781(94)90249-6 Kudo, 1995, Tissue-specific transcriptional regulation of human leukosialin (CD43) gene is achieved by DNA methylation, J Biol Chem, 270, 13298, 10.1074/jbc.270.22.13298 Brenner, 1994, GFAP promoter directs astrocyte-specific expression in transgenic mice, J Neurosci, 14, 1030, 10.1523/JNEUROSCI.14-03-01030.1994 Vile, 1994, A comparison of the properties of different retroviral vectors containing the murine tyrosinase promoter to achieve transcriptionally targeted expression of the HSVtk or IL-2 genes, Gene Ther, 1, 307 He, 2000, The targeted expression of interleukin-2 in human hepatocellular carcinoma cells, J Exp Clin Cancer Res, 19, 183 Lan, 1997, In vivo selective gene expression and therapy mediated by adenoviral vectors for human carcinoembryonic antigen-producing gastric carcinoma, Cancer Res, 57, 4279 Hudson, 1990, Structure and inducible regulation of the human c-erb B2/neu promoter, J Biol Chem, 265, 4389, 10.1016/S0021-9258(19)39577-8 Chen, 1995, Breast cancer selective gene expression and therapy mediated by recombinant adenoviruses containing the DF3/MUC1 promoter, J Clin Invest, 96, 2775, 10.1172/JCI118347