The potential of antisense as a CNS therapeutic

Bibliography

• TANAKA S: [Gene diagnosis of Alzheimer's disease and Parkinson's disease]. Rills& Byori (2002) 50(10):965–969. Japanese.
   Google Scholar
• WAKUTANI Y, KOWA H, KUSUMI M et al: Genetic analysis of vascular factors in Alzheimer's disease. Ann. NY Acad. Sci. (2002) 977:232–238.
   PubMed Web of Science ®Google Scholar
• PARDRIDGE WM: Why is the global CNS pharmaceutical market so under-penetrated? Drug Discov. Today (2002) 7(1):5–7.
   Google Scholar
• MILLER G: Breaking down barriers. Science (2002) 297(5589):1116–1118.
   PubMed Web of Science ®Google Scholar
• PARDRIDGE WM: Crossing the blood-brain barrier: are we getting it right? Drug Discov. Today (2001) 6(1):1–2.
   Google Scholar
• TOULME JJ: New candidates for true antisense. Nature Biotechnol (2001) 19(1):17–18.
   PubMed Web of Science ®Google Scholar
• CROOKE S: Molecular mechansims of action of antisense drugs. Biochlin. Biophys. Acta (1999) 1489(1):31–44.
   PubMed Web of Science ®Google Scholar
• CHIANG M, CHAN H, ZOUNES M et al: Antisense oligonucleotides inhibit intercellular adhesion molecule 1 expression by two distinct mechanisms. j Biol. Chem. (1991) 266(27):18162–18171.
   PubMed Web of Science ®Google Scholar
• VAN HUIJSDUIJNEN RH,BOMBRUN A, SWINNEN D:Selecting protein tyrosine phosphatases as drug targets. DDT(2002) 7(19):1013–1019.
   PubMed Web of Science ®Google Scholar
• ZINKER BA, RONDINONE CM, TREVILLYAN JM et al: PTP1B antisense oligonucleotide lowers PTP1B protein, normalizes blood glucose, and improves insulin sensitivity in diabetic mice. Proc. Nati Acad. Li. USA (2002) 99(17):11357–11362.
   PubMed Web of Science ®Google Scholar
• GUM RJ, GAEDE LL, KOTERSKI SL et al.: Reduction of protein tyrosine phosphatase 1B increases insulin-dependent signaling in obiob mice. Diabetes (2003) 52(1):21–28.
   PubMed Web of Science ®Google Scholar
• DOVE A: Antisense and sensibility. Nature Biotechnol (2002) 20:121–124.
   PubMed Web of Science ®Google Scholar
• ••Good overview of technological hurdles beingovercome in the field of antisense research.
   Google Scholar
• MIR KU, SOUTHERN EM:Determining the influence of structure on hybridization using oligonucleotide arrays. Nature Biotechnol (1999) 17:788–792.
   PubMed Web of Science ®Google Scholar
• DINGY, LAWRENCE CE: Statistical prediction of single-stranded regions inRNA secondary structure and application to predicting effective antisense target sites and beyond. Nucleic Acids Res. (2001) 29(5):1034–1046.
   PubMed Web of Science ®Google Scholar
• MILNER N, MIR KU, SOUTHERN EM: Selecting effective antisense reagents on combinatorial oligonucleotide arrays. Nature Biotechnol (1997) 15:537–541.
   PubMed Web of Science ®Google Scholar
• VICKERS TA, WYATT JA, FREIER SM:Effects of RNA secondary structure on cellular anitsense activity. Nucleic Acids Res. (2000) 28(6):1340–1347.
   PubMed Web of Science ®Google Scholar
• SHCHEPINOV MS, CASE-GREEN SC, SOUTHERN EM: Steric factors influencing hybridisation of nucleic acids to oligonucleotide arrays. Nucleic. Acids Res. (1997) 25(6):1155–1162.
   PubMed Web of Science ®Google Scholar
• BELTINGER C, SARAGOVI H,SMITH R et al: Binding, uptake, and intracellular trafficking of phosphorothioate-modified oligodeoxynucleotides. Clin. Invest. (1995) 95:1814–1823.
   Google Scholar
• SAZANI P, KANG SH, MATER MA et al: Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs. Nucleic Adds Res. (2001) 29(19):3965–3974.
   PubMed Web of Science ®Google Scholar
• BRAASCH DA, COREY DR:Novel antisense and peptide nucleic acidstrategies for controlling gene expression. Biochemistry (2002) 41(14):4503–4510.
   PubMed Web of Science ®Google Scholar
• MORITA K, HASEGAWA C, KANEKO M et al.: 2 '-0,4 --C-ethylene-bridged nucleic acids (ENAs): highly nuclease-resistant thermodynamically stable oligonucleotides for antisense drug. Bioorg. Med. Chem. Lett. (2002) 7(12):73–76.
   Google Scholar
• KURRECK J, WYSZKO E, GILLEN C, ERDMANN VA: Design of antisense oligonucleotides stabilised by locked nucleic acids. Nucleic Acids Res. (2002) 30(9):1911–1918.
   PubMed Web of Science ®Google Scholar
• HUGHES M, HUSSAIN M, NAWAZ Q, SAYYED P, AKHTAR S: The cellular delivery of antisense oligonucleotides and ribozymes. Drug Discov. Today (2001) 6(6):303–315.
   PubMed Web of Science ®Google Scholar
• ••Reviews a wide-range of AS-ODN deliveryvehicles for both in vitro and in vivo use.
   Google Scholar
• ESTIBEIRO P, GODFRAY J: Antisense as a neuroscience tool and therapeutic agent. Trends Neurosci. (2001) 24(10:556–562.
   Google Scholar
• ••Reviews the use of antisense both as aresearch tool and a therapeutic agent in the nervous system.
   Google Scholar
• PRZEWLOCKA B, SIEJA A,STAROWICZM K et al.: Knockdown of spinal opioid receptors by antisense targeting 13-arrestin reduces morphine tolerance and allodynia in rat. Neuracci Lett. (2002) 325:107–110.
   Google Scholar
• YUE S, LUO Z, FENG D: Protective effect of c-fos antisense oligonucleotides on brain damage induced by glutamate. Zhonghua Yi Xue Za(2001) 83(3):145–149.
   Google Scholar
• BIN SUN H, RUAN Y, YOKOTA H: Involvement of the calcium-independent receptor for a-latrotoxin in brain ischemia. Brain Res. Mol. Brain Res. (2002) 104(2):246.
   PubMedGoogle Scholar
• KUMAR V, FARR S, FLOOD J et al: Site-directed antisense oligonucleotide decreases the expression of amyloid precursor protein and reverses deficits in learning and memory in aged SAMP8 mice. Peptides (2000) 21(12):1769–1775.
   PubMed Web of Science ®Google Scholar
• MACDONALD TJ, LADISCH S: Antisense to integrin a v inhibits growth and induces apoptosis in medulloblastoma cells. Anticancer Res. (2001) 21:3785–3792.
   PubMed Web of Science ®Google Scholar
• CLARK CL, CECIL PK, SINGH D, GRAY DM: CD, absorption and thermodynamic analysis of repeating dinucleotide DNA, RNA and hybrid duplexes [d/r(AC)]12.rd/r(GT/U)112 and the influence of phosphorothioate substitution. Nucleic Acids Res. (1997) 25(20):4098–4105.
   Google Scholar
• KHATSENKO 0, MORGAN R, TRUONG L et aL: Absorption of antisense oligonucleotides in rat intestine: effect of chemistry and length. Antisense Nucleic Add Drug Dev. (2000) 10(1):35–44.
   PubMedGoogle Scholar
• GEARY RS, WATANABE TA, TRUONG L et al.: Pharmacokineticproperties of 2'( 2 methoxyethy0 -modified oligonucleotide analogues in rats. Pharinacol. Exp. Mei (2001) 296(3):890–897.
   PubMed Web of Science ®Google Scholar
• SUMMERTON J, WELLER D: Morpholino antisense oligomers: design, preparation, and properties. Antisense Nucleic Add Drug Dev. (1997) 7(3):187–195.
   PubMedGoogle Scholar
• WAHLESTEDT C, SALMI P, GOOD L et al.: Potent and nontoxic antisense oligonucleotides containing locked nucleic acids. Proc. Natl. Acad. Li. USA (2000) 97(10):5633–5638.
   PubMed Web of Science ®Google Scholar
• MCMAHON BM, STEWART JA, JACKSON J et al: Intraperitoneal injection of antisense peptide nucleic acids targeted to the p receptor decreases response to morphine and receptor protein levels in rat brain. Brain Res. (2001) 904(2):345–349.
   PubMed Web of Science ®Google Scholar
• TYLER BM, JANSEN K,MCCORMICK DJ et al.: Peptide nucleic acids targeted to the neurotensin receptor and administered i.p. cross the blood-brain barrier and specifically reduce gene expression. Proc. Natl. Acad. Sci. USA (1999) 96(12):7053–7058.
   Google Scholar
• BANKS WA, FARR SA, BUTT W et al: Delivery across the blood-brain barrier of antisense directed against amyloid13: reversal of learning and memory deficits in mice overexpressing amyloid precursor protein. J. Pharmacol Exp. Ther. (2001) 297(3):1113–1121.
   PubMed Web of Science ®Google Scholar
• ROBINSON ESJ, NUTT DJ, JACKSON HC, HUDSON AL: Antisense oligonucleotides in psychopharmacology and behaviour: promise and pitfalls. I Psychopharmacol (1997) 11(3):259–269.
   PubMed Web of Science ®Google Scholar
• ••An excellent review.
   Google Scholar
• PARDRIDGE WM: Brain drug targetingand gene technologies. fpn. Pharmacol (2001) 87(2):97–103.
   Web of Science ®Google Scholar
• MANOHARAN M: Oligonucleotide conjugates as potential antisense drugs with improved uptake, biodistribution, targeted delivery, and mechanism of action. Antisense Nucleic Acid Drug Dev (2002) 12:103–128.
   PubMedGoogle Scholar
• READ TA, THORSEN F, BJERKVIG R: Localised delivery of therapeutic agents to CNS malignancies: old and new approaches. Curl: Pharm. Biotechnoi (2002) 3(3):257–273.
   PubMedGoogle Scholar
• ROH H, PIPPIN J, DREBIN JA: Down-regulation of HER2/neu expression induces apoptosis in human cancer cells that overexpress HER2/neu. Cancer Res. (2000) 60:560–565.
   PubMed Web of Science ®Google Scholar
• MUKAI S, KONDO Y, KOGA S et al.: 2-5A antisense telomerase RNA therapy for intracranial malignant gliomas. Cancer Res. (2000) 60:4461–4467.
   PubMed Web of Science ®Google Scholar
• LAKKARAJU A, DUBINSKI JM, LOW WC, RAHMAN Y: Neurons are protected from excitotoxic death by p53 antisense oligonucleotides delivered in anionic liposomes../. Bid. Chem. (2001) 276(34):32000–32007.
   Google Scholar
• SHI N, PARDRIDGE WM: Noninvasive gene targeting to the brain. Proc. Natl. Acad. Sci. USA (2000) 97(13):7567–7572.
   PubMed Web of Science ®Google Scholar
• ZHANG Y, JEONG LH, BOADO RJ, PARDRIDGE WM: Receptor-mediated delivery of an antisense gene to human brain cancer cells. J. Gene Med. (2002) 4(2):183–194.
   PubMed Web of Science ®Google Scholar
• •Demonstrates the potential of PILs as delivery vehicles that can carry DNA (in this case an antisense gene) across the BBB.
   Google Scholar
• ZHANG Y, ZHU C, PARDRIDGE WM: Antisense gene therapy of brain cancer with an artificial virus gene delivery system. Md. Ther (2002) 6(1):67–72.
   PubMed Web of Science ®Google Scholar
• CLEEK RL, REGE AA, DENNER LA, ESKIN SG, MIKOS AG: Inhibition of smooth muscle cell growth in vitro by an antisense oligodeoxynucleotide released from poly(DL-lactic-co-glycolic acid) microparticles. Biomed. Mater Res. (1997) 35(4):525–530.
   PubMed Web of Science ®Google Scholar
• KHAN A, SOMMER W, FUXE K, AKHTAR S: Site-specific administration of antisense oligonucleotides using biodegradable polymer microspheres provides sustained delivery and improved subcellular biodistribution in the neostriatum of the rat brain. I Drug Target. (2000) 8(5):319–334.
   PubMed Web of Science ®Google Scholar
• EPA WR, GREFERATH U, SHAFTON A et al.: Downregulation of the p75 neurotrophin receptor in tissue culture and in vivo, using 13-cyclodextrin-adamantane-oligonucleotide conjugates. Antisense Nucleic Add Drug Dev (2000) 10(6):469–478.
   PubMedGoogle Scholar
• BIELINSKA A,KUKOWSKA-LATALLO JF, JOHNSON J, TOMALIA DA, BAKER JR Jr: Regulation of in vitro gene expression using antisense oligonucleotides or antisense expression plasmids transfected using starburst PAMAM dendrimers. Nucleic Acids Res. (1996) 24(11):2176–2182.
   PubMed Web of Science ®Google Scholar
• Y00 H, JULIANO RL: Enhanced delivery of antisense oligonucleotides with fluorophore-conjugated PAMAM dendrimers. Nucleic Acids Res. (2000) 28(21):4225–4231.
   PubMed Web of Science ®Google Scholar
• NORMAND-SDIQUI N, SAGHIR A: Oligonucleotide delivery: uptake of rat transferrin receptor antibody (0X-26) conjugates into an in vitro immortalised cell line model of the blood-brain barrier. Int. J. Pim= (1998) 163:63–71.
   Web of Science ®Google Scholar
• SHI N, BOADO RJ, PARDRIDGE WM: Antisense imaging of gene expression in the brain in vivo. Proc. Nati Acad. Sci. USA (2000) 97(26):14709–14714.
   PubMed Web of Science ®Google Scholar
• •Demonstration of an AS-PNA being delivered across the BBB (conjugated to 0X26 monoclonal antibody).
   Google Scholar
• KOPPELHUS U, AWASTHI SK, ZACHAR V et al.: Cell-dependent differential cellular uptake of PNA, peptides,and PNA-peptide conjugates. Antisense Nucleic Acid Drug Dev (2002) 12:51–63.
   PubMedGoogle Scholar
• SHOICHET MS, WINN SR: Cell delivery to the central nervous system. Adv. Drug Deify. Rev (2000) 42(1-2):81–102.
   PubMed Web of Science ®Google Scholar
• BAKHSHI S, NORTH RB: Implantable pumps for drug delivery to the brain. Neurooncol (1995) 26(2):133–139.
   PubMed Web of Science ®Google Scholar
• FISHER RS, HO J: Potential new methods for antiepileptic drug delivery. CNS Drugs (2002) 16(9):579–593.
   PubMed Web of Science ®Google Scholar
• GROOTHUIS DR: The blood-brain and blood-tumor barriers: a review of strategies for increasing drug delivery. Neurooncology (2000) 2(1):45–59.
   PubMed Web of Science ®Google Scholar
• DELONG R, STEPHENSON K, LOFTUS T et al: Characterization of complexes of oligonucleotides with polyamidoamine starburst dendrimers and effects on intracellular delivery. Pharm. Sci. (1997) 86(6):762–764.
   PubMed Web of Science ®Google Scholar
• PARDRIDGE WM: Drug and gene targeting to the brain with molecular Trojan horses. Nat. Rev. Drug Discov (2002) 1(2):131–139.
   PubMed Web of Science ®Google Scholar
• •Briefly reviews some of the issues surrounding the delivery ofCNS therapeutics across the BBB.
   Google Scholar
• ARNER S, MEYERSON BA: Lack of analgesic effect of opioids on neuropathic and idiopathic forms of pain. Path (1988) 33(1):11–23.
   Google Scholar
• BOHN LM, GAINETDINOV RR, LIN FT, LEFKOWITZ RJ, CARON MG: ff-opioid receptor desensitization by 13-arrestin-2 determines morphine tolerance but not dependence. Nature (2000) 408(6813):720–723.
   PubMed Web of Science ®Google Scholar
• FRANTSEVA MV, KOKAROVTSEVA L, PEREZ VELAZQUEZ JL: Ischemia-induced brain damage depends on specific gap-junctional coupling. Cereb. Blood Flow Metab. (2002) 22(4):453–462.
   PubMed Web of Science ®Google Scholar
• JAIN K: Pharmacogenomics. Appl. Neurogenomics (2001) 2(2):143–152.
   Google Scholar
• KECK ME, HOLSBOER F: Hyperactivity of CRH neuronal circuits as a target for therapeutic intervention in affective disorders. Peptides (2001) 22:835–844.
   PubMed Web of Science ®Google Scholar
• SKUTELLA T, MONTKOWSKI A, STOHR T et al.: Corticotropin-releasing hormone (CRH) antisense oligodeoxynucleotide treatment attenuates social defeat-induced anxiety in rats. Mol. Neurobid. (1994) 14(5):579–588.
   PubMed Web of Science ®Google Scholar
• MERCATANTE DR, SAZANI P, KOLE R: Modification of alternative splicing by antisense oligonucleotides as a potential chemotherapy for cancer and other diseases. Curr. Cancer Drug Targets (2001) 1(3):211–230.
   PubMed Web of Science ®Google Scholar
• MERCATANTE DR, KOLE R:Control of alternative splicing by antisense oligonucleotides as a potential chemotherapy: effects of gene expression. Biochim. Biophys. Acta (2002) 1597:126–132.
   Google Scholar
• FRIEDMAN KJ, KOLE J, COHN JA et al: Correction of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides. J. Biol. Chem. (1999) 274(51):36193–36199.
   PubMed Web of Science ®Google Scholar
• SLAUGENHAUPT SA,BLUMENFELD A, GILL SP et al.: Tissue-specific expression of a splicing mutation in the IKBKAP gene causesfamilial dysautonomia. Am. I Hum. Genet. (2001) 68:598–605.
   PubMed Web of Science ®Google Scholar
• HALFORD JC: Pharmacology of appetite suppression: implication for the treatment of obesity. Curr. Drug Targets (2001) 2 (4) :353–370.
   PubMed Web of Science ®Google Scholar
• HEISLER LK, COWLEY MA,TECOTT LH et al.: Activation of central melanocortin pathways by fenfluramine. Science (2002) 297(5581):609–611.
   Google Scholar
• KRICHEVSKY AM, KOSIK KS: RNAi functions in cultured mammalian neurons. Proc. Nati Acad. Sci USA (2002) 99(18):11926–11929.
   PubMed Web of Science ®Google Scholar
• PAGED, ESTIBEIRO P: Acute gene knockdown: antisense and RNAi as tools and therapies. Recent Res. Dev Biochem. Submitted.
   Google Scholar
• HANNON GJ: RNA interference. Nature (2002) 418(6894):244–251.
   PubMed Web of Science ®Google Scholar
• YAZAKI T, AHMAD S, CHAHLAVI A et al.: Treatment of glioblastoma U-87 by systemic administration of an antisense protein kinase C-a phosphorothioate oligodeoxynucleotide. Mol. Pharmacol (1996) 50:236–242.
   PubMed Web of Science ®Google Scholar
• MCMAHON BM, STEWART J, FAUQ A eta].: Using peptide nucleic acids as gene-expression modifiers to reduce 13-amyloid levels. Mol. Neurosci. (2002) 19:71–76.
   Google Scholar