Regulus Btn

Print Version


The following is a listing of some articles of interest.

New. Hannis JC, Manalili SM, Hall TA, Ranken R, White N, Sampath R, Blyn LB, Ecker DJ, Mandrell RE, Fagerquist CK, Bates AH, Miller WG, Hofstadler SA. High resolution genotyping of campylobacter using PCR and high-throughput mass spectrometry. J Clin Microbiol. 2008 February 13;[Epub ahead of print].

New. Yamaguchi K, Yang L, McCall S, Huang J, Yu XX, Pandey SK, Bhanot S, Monia BP, Li YX, Diehl AM. Diacylglycerol acyltransferase 1 anti-sense oligonucleotides reduce hepatic fibrosis in mice with nonalcoholic steatohepatitis. Hepatology. 2008 February;47(2):625-635.

New. Graff JR, Konicek BW, Konicek W, Carter JH, Marcusson EG. Targeting the Eukaryotic Translation Initiation Factor 4E for Cancer Therapy. Cancer Res. 2008 February;68(3):631-634.

New. Yu XX, Pandey SK, Booten SL, Murray SF, Monia BP, Bhanot S. Reduced adiposity and improved insulin sensitivity in obese mice with antisense suppression of 4E-BP2 expression. Am J Physiol Endocrinol Metab. 2008 Jan 15;[Epub ahead of print].

New. Esau CC. Inhibition of microRNA with antisense oligonucleotides. PMID. 2008 Jan;44(1):55-60.

New. Tillman LG, Geary RS, Hardee GE. Oral delivery of antisense oligonucleotides in man. J Pharm Sci. 2008 Jan;97(1):225-236.

Antisense Pharmacology - Proof of Mechanism and Mechanisms of Action

General Antisense Review:

Dean, N.M. Functional genomics and target validation approaches using antisense oligonucleotides technology. Curr Opin Biotechnol. 12(6):622-5, 2001.

Geary, R.S., Yu, R.Z., Levin, A.A. Pharmacokinetics of phosphorothioate antisense oligodeoxynucleotides. Curr Opin Investig Drugs. 2(4):562-573, 2001.

Crooke, S.T. Progress in antisense technology: The end of the beginning. Methods Enzymol. 313(Antisense Technology, Part A):3-45, 2000.

Crooke, S.T. Antisense Therapeutics. Biotechnol Genet Eng Rev. 1998;15:121-57.


Antisense Oligonucleotides- Mechanism of Action

Vickers, T.A., Koo, S., Bennett, C.F., Crooke, S.T., Dean, N.M., Baker, B.F. Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents. A comparative analysis. J Biol. Chem. 278(9):7108-18, 2003.

Sazani, P., Gemignani, F., Kang, S-H, Maier, M.A., Manoharan, M. Persmark, M., Bortner, D., and Kole, R. Systematically delivered antisense oligomers upregulate gene expression in mouse tissues. Nat. Biotechnol. 20: 1228-1233, 2002.

Karras, J. G., McKay, R. A., Dean, N. M., and Monia, B. P. Deletion of individual exons and induction of soluble murine interleukin-5 receptor-alpha chain expression through antisense oligonucleotide-mediated redirection of pre-mrna splicing. Mol Pharmacol. 58(2):380-387, 2000.

Crooke, S.T. Molecular mechanisms of action of antisense drugs. Biochim Biophys Aca.1489 (1): 31-43, 1999.

Taylor, J. K., Zhang, Q. Q., Wyatt, R. and Dean, N. M. Induction of endogenous Bcl-xS through the control of Bcl-x pre-mRNA splicing by antisense oligonucleotides. Nature Biotechnology.17(11):1097-1100, 1999.

Wu, H., Lima, W.F. and Crooke, S.T. Properties of cloned and expressed human RNase H. J. Biol. Chem. 274(40)28270-28278, 1999.

Baker, B. F., Lot, S. S., Condon, T. P., Cheng-Flournoy, S., Lesnik, E. A., Sasmor, H. M., and Bennett, C. F. 2'-o-(2-methoxy)ethyl-modified anti-intercellular adhesion molecule 1 (icam-1) oligonucleotides selectively increase the icam-1 mrna level and inhibit formation of the icam-1 translation initiation complex in human umbilical vein endothelial cells. Journal of Biological Chemistry. 272(18):11994-12000, 1997.


Antisense Oligonucleotides as a Gene Functionalization Tool

Esau CC. Inhibition of microRNA with antisense oligonucleotides. PMID. 2008 Jan;44(1):55-60.

Waring, J.F., Ciurlionis, R., Clampit, J.E., Morgan, S., Gum, R.J., Jolly, R.A., Kroeger, P., Frost, L., Trevillyan, J., Zinker, B.A., Jirousek, M., Ulrich, R.G., Rondinone, C.M. PTP1B antisense-treated mice show regulation of genes involved in lipogenesis in liver and fat. Molecular and Cellular Endocrinology. 203: 155-168, 2003.

Choi, Y. Zhang, J., Murga, C. Yu, H., Koller, E., Monia, B.P., Gutkind, J.S., and Li, W. PTEN, but not SHIP and SHIP2, suppresses the PI3K/Akt pathway and induces growth inhibition and apoptosis of myeloma cells. Oncogene. 21:(34)5289-300, 2002.

Niu, G., K.L. Wright, M. Huang, L. Song, E. Haura, J. Turkson, S. Zhang, T. Wang, D. Sinibaldi, D. Coppola, R. Heller, L.M. Ellis, J. Karras, J. Broomberg, D. Pardoll, R. Jove, and H. Yu. Constitutive stat3 activity up-regulates VEGF expression and tumor angiogenesis. Oncogene. 2002 Mar 27;21(13):2000-8.

Potapova, O., Anisimov, S.V., Gorospe, M., Dougherty, R.H., Gaarde, W.A., Boheler, K.R., Holbrook, N.J. Targets of c-Jun NH(2)-terminal Kinase 2-mediated Tumor Growth Regulation Revealed by Serial Analysis of Gene Expression. Cancer Res. 62(11):3257-3263, 2002.

Bulavin, D.V., Higashimoto, Y., Popoff, I.J., Gaarde, W.A., Basrur, V., Potapova, O. Appella, E., and Fornace, Jr., A.J. Initiation of a G2/M checkpoint after ultraviolet radiation requires p38 kinase. Nature. 2001 May 3;411(6833):102-7.

Urban, G., Golden, T., Aragon, I. V., Scammell, J. G., Dean, N. M., and Honkanen, R. E. Identification of an estrogen-inducible phosphatase (pp5) that converts mcf-7 human breast carcinoma cells into an estrogen-independent phenotype when expressed constitutively. J Biol Chem. 276(29):27638-27646, 2001.

Li, F., Ackermann, E.J., Bennett, C.F., Rothermel, A.L., Plescia, J., Tognin, S., Villa, A., Marchisio, P.C., and Altieri, D.C. Pleiotropic cell-division defects and apoptosis induced by interference with survivin function. Nature Cell Biol. 1:461-466, 1999.


Antisense Drug Discovery

Asthma

Crosby JR, Guha M, Tung D, Miller DA, Bender B, Condon TP, York-Defalco C, Geary RS, Monia BP, Karras JG, Gregora SA. Inhaled CD86 Antisense Oligonucleotide Suppresses Pulmonary Inflammation and Airway Hyper-responsiveness in Allergic Mice. J Pharmacol Exp Ther. 2007 Mar 26; [Epub ahead of print].

Karras, J. G., McGraw, K., McKay, R. A., Cooper, S. R., Lerner, D., Lu, T., Walker, C., Dean, N. M., and Monia, B. P. Inhibition of antigen-induced eosinophilia and late phase airway hyperresponsiveness by an il-5 antisense oligonucleotide in mouse models of asthma. J Immunol. 164(10):5409-5415, 2000.


Cardiovascular

Graham MJ, Lemonidis KM, Whipple CP, Subramaniam A, Monia BP, Crooke ST, Crooke RM. Antisense Inhibition of Proprotein Convertase Subtilisin Kexin 9 Reduces Serum LDL in Hyperlipidemic Mice. J Lipid Res. 2007 Jan 24.

Zhang YL, Hernandez-Ono A, Siri P, Weisberg S, Conlon D, Graham MJ, Crooke RM, Huang LS, Ginsberg HN. Aberrant Hepatic Expression of PPAR{gamma}2 Stimulates Hepatic Lipogenesis in a Mouse Model of Obesity, Insulin Resistance, Dyslipidemia, and Hepatic Steatosis. J Biol Chem. 2006 Dec 8;281(49):37603-15.

Kastelein JJ, Wedel MK, Baker BF, Su J, Bradley JD, Yu RZ, Chuang E, Graham MJ, Crooke RM. Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation. 2006 Oct 17;114(16):1729-35.

Burnett JR. Drug evaluation: ISIS-301012, an antisense oligonucleotide for the treatment of hypercholesterolemia. Curr Opin Mol Ther. 2006 Oct;8(5):461-7.

Bell TA 3rd, Brown JM, Graham MJ, Lemonidis KM, Crooke RM, Rudel LL. Liver-specific inhibition of acyl-coenzyme a:cholesterol acyltransferase 2 with antisense oligonucleotides limits atherosclerosis development in apolipoprotein B100-only low-density lipoprotein receptor-/- mice. Arterioscler Thromb Vasc Biol. 2006 Aug;26(8):1814-20.

Crooke RM, Graham MJ, Lemonidis KM, Whipple CP, Koo S, Perera RJ. An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis. J. Lipid Res. 2005; 46: 872-884.


Metabolic

Yamaguchi K, Yang L, McCall S, Huang J, Yu XX, Pandey SK, Bhanot S, Monia BP, Li YX, Diehl AM. Diacylglycerol acyltransferase 1 anti-sense oligonucleotides reduce hepatic fibrosis in mice with nonalcoholic steatohepatitis. Hepatology. 2008 February;47(2):625-635.

Yu XX, Pandey SK, Booten SL, Murray SF, Monia BP, Bhanot S. Reduced adiposity and improved insulin sensitivity in obese mice with antisense suppression of 4E-BP2 expression. Am J Physiol Endocrinol Metab. 2008 Jan 15;[Epub ahead of print].

Pandey SK, Yu XX, Watts LM, Michael MD, Sloop KW, RIvard AR, Leedon TA, Manchem VP, Samadzadeh L, McKay RA, Monia BP, Bhanot S. Reduction of Low-Molecular-Weight Protein Tyrosine Phosphatase Expression Improves Hyperglycemia and Insulin Sensitivity in Obese Mice. J Bio Chem. 2007 Mar 12; [Epub ahead of print].

Samuel VT, Liu ZX, Wang A, Beddow SA, Geisler JG, Kahn M, Zhang XM, Monia BP, Bhanot S, Shulman GI. Inhibition of Protein Kinase Cepsilon Prevents Hepatic Insulin Resistance in Nonalcoholic Fatty Liver Disease. J Clin Invest. 2007 Mar 1;117(3):739-745.

Samuel VT, Choi CS, Phillips TG, Romanelli AJ, Geisler JG, Bhanot S, McKay R, Monia B, Shutter JR, Lindberg RA, Shulman GI, Veniant MM. Targeting foxo1 in mice using antisense oligonucleotide improves hepatic and peripheral insulin action. Diabetes. 2006 Jul;55(7):2042-50.

Esau C, Davis S, Murray SF, Yu XX, Pandey SK, Pear M, Watts L, Booten SL, Graham M, McKay R, Subramaniam A, Propp S, Lollo BA, Freier S, Bennett CF, Bhanot S, Monia BP. miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting.
Cell Metab. 2006 Feb;3(2):87-98.

Yu XX, Murray SF, Pandey SK, Booten SL, Bao D, Song XZ, Kelly S, Chen S, McKay R, Monia BP, Bhanot S. Antisense oligonucleotide reduction of DGAT2 expression improves hepatic steatosis and hyperlipidemia in obese mice.Hepatology. 2005 Aug;42(2):362-71.

Butler, M., McKay, R.A., Popoff, I.J., Gaarde, W.A., Witchell, D., Murray, S.F., Dean, N.M., Bhanot, S., and Monia, B.P. Specific inhibition of PTEN expression reverses hyperglycemia in diabetic mice. Diabetes. 51(4):1028-34, 2002.

Rondinone, C.M., Trevillyan, J.M., Clampit, J., Gum, R.J., Berg, C., Kroeger, P., Frost, L., Zinker, B.A., Reilly, R., Ulrich, R., Butler, M., Monia, B.P., Jirousek, M.R., and Waring, J.F. Protein tyrosine phosphatase 1B reduction regulates adiposity and expression of genes involved in lipogenesis. Diabetes. 2002 Aug;51(8):2405-11.

Zinker, B.A., Rondinone, C.M., Trevillyan, J.M., Gum, R.J., Clampit, J.E., Waring, J.F., Xie, N., Wilcox, D., Jacobson, P., Frost, L., Kroeger, P.E., Reilly, R.M., Koterski, S., Opgenorth, T.J., Ulrich, R.G., Crosby, S., Butler, M., Murray, S.F., McKay, R.A., Bhanot, S., Monia, B.P., and Jirousek, M.R. PTP1B antisense oligonucleotides lowers PTP1B protein, normalizes blood glucose, and improved insulin sensitivity in diabetic mice. Proc. Natl. Acad. Sci. USA 99 (17): 11357-11362, 2002.


Inflammatory Diseases

Myers, K.J., Murthy, S., Flanigan, A., Witchell, D.R., Butler, M., Murray, S., Siwkowski, A., Goodfellow, D., Madsen, K., Baker, B. Antisense oligonucleotide blockade of tumor necrosis factor-alpha in two murine models of colitis. J Pharmacol Exp Ther. 304(1):411-424, 2003.

Bowen-Yacyshyn, M.B., Bennett, C.F., Nation, N., Raynor, D., and Yacyshyn, B.R. Amelioration of chronic and spontaneous intestinal inflammation with an antisense oligonucleotide (ISIS 9125) to ICAM-1 in the HLA-B27/2 microglobulin transgenic rat model. J Pharmacol Exp Ther. 2002 Sep;302(3):908-17.

Bennett, C. F., Kornbrust, D., Henry, S., Stecker, K., Howard, R., Cooper, S., Dutson, S., Hall, W., and Jacoby, H. I. An icam-1 antisense oligonucleotide prevents and reverses dextran sulfate sodium-induced colitis in mice. J Pharmacol Exp Ther. 280(2):988-1000, 1997.


Oncology

Graff JR, Konicek BW, Konicek W, Carter JH, Marcusson EG. Targeting the Eukaryotic Translation Initiation Factor 4E for Cancer Therapy. Cancer Res. 2008 February;68(3):631-634

Graff JR, Konicek BW, Vincent TM, Lynch RL, Monteith D, Weir SN, Schwier P, Capen A, Goode RL, Dowles MS, Chen Y, Zhang H, Sissons S, Cox K, McNulty AN, Parsons SH, Wang T, Sams L, Geeganage S, Douglass LE, Neubauer BL, Dean NM, Blanchard K, Shou J, Stancato LF, Carter JH, Marcusson EG. Therapeutic Suppression of Translation Initiation Factor eIF4E Expression Reduces Tumor Growth Without Toxicity. J Clin Invest. 2007 September;117:2638-2648.

Kim Y, Yoon JW, Xiao X, Dean NM, Monia BP, Marcusson EG. Selective Down-regulation of Glioma-associated Oncogene 2 Inhibits the Proliferation of Hepatocellular Carcinoma Cells. Cancer Res. 2007 Apr 15;67(8):3583-93.

Dagvadorj A, Collins S, Jomain JB, Abdulghani J, Karras J, Zellweger T, Li H, Nurmi M, Alanen K, Mirtti T, Visakorpi T., Budendorf L, Goffin V, Nevalainen MT. . Autocrine Prolactin Promotes Prostate Cancer Cell Growth via Jak2-Stat5a/b Signaling Pathway. Endocrinology. 2007 Apr 5; [Epub ahead of print].

Koller E, Propp S, Zhang H, Zhao C, Xiao X, Chang M, Hirsch SA, Shepard PJ, Koo S, Murphy C, Glazer RI, Dean NM. Use of a Chemically Modified Antisense Oligonucleotide Library to Identify and Validate Eg5 (Kinesin-Like 1) as a Target for Antineoplastic Drug Development. Cancer Res. 2006 Feb 15;66(4):2059-2066.

Chi KN, Eisenhauer E, Fazli L, Jones EC, Goldenberg SL, Powers J, Tu D, Gleave ME. A phase I pharmacokinetic and pharmacodynamic study of OGX-011, a 2'-methoxyethyl antisense oligonucleotide to clusterin, in patients with localized prostate cancer. J Natl Cancer Inst. 2005 Sep 7;97(17):1287-96.

Yang, Y.M., Bost, F., Charbono, W., Dean, N., McKay, R., Rhim, J.S., Depatie, C., Mercola, D. C-Jun NH(2)-terminal kinase mediates proliferation and tumor growth of human prostate carcinoma. Clin Cancer Res. 2003 Jan;9(1):391-401.

Pedersen, I.M., Kitada, S., Leoni, L.M., Zapata, J.M., Karras, J.G., Tsukada, N., Kipps, T.J., Choi, Y.S., Bennett, F., Reed, J.C. Protection of CLL B cells by a follicular dendritic cell line is dependent on induction of Mcl-1. Blood. 2002 Sep 1;100(5):1795-801.

Fennell, D.A., Corbo, M.V., Dean, N.M., Monia, B.P., and Cotter, F.E. In Vivo Expression of bcl-xl expression facilitates chemotherapy-induced leukemia cell death in a scid/nod-hu model. Br J Haematol. 112(3): 706-713, 2001.

Bulavin, D.V., Higashimoto, Y., Popoff, I.J., Gaarde, W.A., Basrur, V., Potapova, O. Appella, E., and Fornace, Jr., A.J. Initiation of a G2/M checkpoint after ultraviolet radiation requires p38 kinase. Nature. 2001 May 3;411(6833):102-7.

Mesri, M., Morales-Ruiz, M., Ackermann, E. J., Bennett, C. F., Pober, J. S., Sessa, W. C., and Altieri, D. C. Suppression of vascular endothelial growth factor-mediated endothelial cell protection by survivin targeting. Am J Pathol. 158(5):1757-1765, 2001.

Zellweger, T., Miyake, H., Cooper, S., Chi, K., Conklin, B.S., Monia, B.P., and Gleave, M.E. Antitumor activity of antisense clusterin oligonucleotides is improved in vitro and in vivo by incorporation of 2'-o-(2-methoxyethyl). J Pharmacol Exp Ther. 298(3): 934-940, 2001.

Li, F., Ackermann, E.J., Bennett, C.F., Rothermel, A.L., Plescia, J., Tognin, S., Villa, A., Marchisio, P.C., and Altieri, D.C. Pleiotropic cell-division defects and apoptosis induced by interference with survivin function. Nature Cell Biol. 1:461-466, 1999.


Anti-viral

Detrick, B., Nagineni, C.N., Grillone, L.R., Anderson, K.P., Henry, S.P., and Hooks, J.J. Inhibition of human cytomegalovirus replication in human retinal epitheal cell model by antisense oligonucleotide. Invest Ophthalmol Vis Sci. 2001 Jan;42(1):163-9.

Zhang, H., Cook, J., Nickel, J., Yu, R., Stecker, K., Myers, K., and Dean, N. M. Reduction of liver fas expression by an antisense oligonucleotide protects mice from fulminant hepatitis. Nat Biotechnol. 18(8):862-867, 2000.

Zhang, H., Hanecak, R., Brown-Driver, V., Azad, R., Conklin, B., Fox, M. C., and Anderson, K. P. Antisense oligonucleotide inhibition of hepatitis c virus (hcv) gene expression in livers of mice infected with an hcv-vaccinia virus recombinant. Antimicrobial Agents and Chemotherapy. 43(2):347-353, 1999.

Hanecak, R., Brown-Driver, V., Fox, M.C., Azad, R.F., Furusako, S. Nozaki, C., Ford, C., Sasmor, H., Anderson, K.P. Antisense oligonucleotide inhibition of hepatitis c virus gene expression in transformed hepatocytes. Journal of Virology. 70(8):5203-5212, 1996.


Central Nervous System

Hua Y, Vickers TA, Baker BF, Bennett CF, Krainer AR. Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the Exon. PLoS Biol. 2007 April;5(4):729-744.

Smith RA, Miller TM, Yamanaka K, Monia BP, Condon TP, Hung G, Lobsiger CS, Ward CM, McAlonis-Downes M, Wei H, Wancewicz EV, Bennett CF, Cleveland DW. Antisense oligonucleotide therapy for neurodegenerative disease. J Clin Invest. 2006 Aug;116(8):2290-6. Epub 2006 Jul 27.


Ocular

Danis R, Criswell M, Orge F, Wancewicz E, Stecker K, Henry S, Monia B. Intravitreous anti-raf-1 kinase antisense oligonucleotide as an angioinhibitory agent in porcine preretinal neovascularization. Curr Eye Res. 2003 Jan;26(1):45-54

Danis RP, Henry SP, Ciulla TA. Potential therapeutic application of antisense oligonucleotides in the treatment of ocular diseases. Expert Opin Pharmacother. 2001 Feb;2(2):277-91


Antisense Pharmacokinetics

Yu RZ, Kim TW, Hong A, Watanabe TA, Gaus HJ, Geary RS. Cross-Species Pharmacokinetic Comparison from Mouse to Man of a Second-Generation Antisense Oligonucleotide, ISIS 301012, Targeting Human Apolipoprotein B-100. Drug Metab Dispos. 2007 Mar;35(3):460-8. Epub 2006 Dec. 15.

Geary, R. S., Khatsenko, O., Bunker, K., Crooke, R., Moore, M., Burckin, T., Truong, L., Sasmor, H., and Levin, A. A. Absolute bioavailability of 2'-o-(2-methoxyethyl)-modified antisense oligonucleotides following intraduodenal instillation in rats. J Pharmacol Exp Ther. 296(3):898-904, 2001.

Geary, R. S., Watanabe, T. A., Truong, L., Freier, S., Lesnik, E. A., Sioufi, N. B., Sasmor, H., Manoharan, M., and Levin, A. A. Pharmacokinetic properties of 2'-o-(2-methoxyethyl)-modified oligonucleotide analogs in rats. J Pharmacol Exp Ther. 296(3):890-897, 2001.

Yu, R. Z., Zhang, H., Geary, R. S., Graham, M., Masarjian, L., Lemonidis, K., Crooke, R., Dean, N. M., and Levin, A. A. Pharmacokinetics and pharmacodynamics of an antisense phosphorothioate oligonucleotide targeting fas mrna in mice. J Pharmacol Exp Ther. 296(2):388-395, 2001.

Butler, M., Crooke, R.M., Graham, M.J., Lemonidis, K.M., Lougheed, M., Murray, S.F., Witchell, D., Steinbrecher, U., Bennet, C.F. Phosphorothioate oligodeoxynucleotides distribute similarly in class a scavenger receptor knockout and wild-type mice. J Pharmacol Exp Ther. 292(2):489-496, 2000.

Graham, M.J., Crooke, S. T., Monteith, D. K., Cooper, S. R., Lemonidis, K. M., Stecker, K. K., Martin, M. J., and Crooke, R. M. In vivo distribution and metabolism of a phosphorothioate oligonucleotide within rat liver after intravenous administration. J Pharmacol. Exp Ther. 286(1):447-458, 1998.

Butler, M., Stecker, K., and Bennett, C. F. Cellular distribution of phosphorothioate oligodeoxynucleotides in normal rodent tissues. Lab Invest. 77(4):379-388, 1997.


Antisense Safety Profile

Levin, A., Henry, S., Monteith, D., and Templin, M. Toxicity of antisense oligonucleotides. Antisense Drug Technology: Principles, Strategies, and Applications (ed. Crooke, S.T.), pp. 201-267. Marcel Dekker, Inc., New York, 2001.

Dorr, A., Glover, J., and Kwoh, J. Clinical safety of phosphorothioate oligodeoxynucleotides. Antisense Drug Technology: Principles, Strategies, and Applications (ed. Crooke, S.T.), pp. 269-290. Marcel Dekker, Inc., New York, 2001.

Henry, S., Stecker, K., Brooks, D., Monteith, D., Conklin, B., and Bennett, C. F. Chemically modified oligonucleotides exhibit decreased immune stimulation in mice. J Pharmacol Exp Ther. 292(2):468-479, 2000.


Antisense Evidence of Clinical Activity

Alicaforsen (ISIS 2302)

Miner PB Jr, Wedel MK, Xia S, Baker BF. Safety and efficacy of two dose formulations of alicaforsen enema compared with mesalazine enema for treatment of mild to moderate left-sided ulcerative colitis: a randomized, double-blind, active-controlled trial. Aliment Pharmacol Ther. 2006 May 15;23(10):1403-13.

Miner PB Jr, Geary RS, Matson J, Chuang E, Xia S, Baker BF, Wedel MK. Bioavailability and therapeutic activity of alicaforsen (ISIS 2302) administered as a rectal retention enema to subjects with active ulcerative colitis. Aliment Pharmacol Ther. 2006 May 15;23(10):1427-34.

van Deventer SJ, Wedel MK, Baker BF, Xia S, Chuang E, Miner PB Jr. A phase II dose ranging, double-blind, placebo-controlled study of alicaforsen enema in subjects with acute exacerbation of mild to moderate left-sided ulcerative colitis. Aliment Pharmacol Ther. 2006 May 15;23(10):1415-25.

Yacyshyn, B.R., Chey, W.Y., Goff, J., Salzberg, B. Baerg, R., Buchman, A.L., Tami, J., Yu, R., Gibiansky, E., and Shanahan, W.R. Double blind, placebo controlled trial of the remission inducing and steroid sparing properties of an ICAM-1 antisense oligodeoxynucleotide, Alicaforsen (ISIS 2302), in active steroid dependent Crohn’s disease. Gut. 51: 30-36, 2002.

Shanahan, W. ISIS 2302, an antisense inhibitor of intercellular adhesion molecule 1 (ICAM-1). In: Antisense Drug Technology: Principles, Strategies, and Applications (ed. Crooke, S.T.), pp. 773-794. Marcel Dekker, Inc., New York, 2001.

Yacyshyn, B.R., Bowen-Yacyshyn, M.B., Jewell, L., Tami, J.A., Bennett, C.F., Kisner, D.L., and Shanahan, Jr., W.R. A placebo-controlled trial of ICAM-1 antisense oligonucleotide in the treatment of Crohn's disease. Gastroenterology. 1998 Jun;114(6):1133-42.


ISIS 104838

Sewell, K.L., Geary, R.S., Baker, B.F., Glover, J.M., Mant, T.G., Yu, R.Z., Tami, J.A., and Dorr, A. Phase I trial of ISIS 104838, a 2'-Methoxyethyl modified antisense oligonucleotide targeting tumor necrosis factor-a. J Pharmacol Exp Ther. 2002 Dec;303(3):1334-43.


Vitravene

Vitravene Study Group. A randomized controlled clinical trial of intravitreous fomivirsen for treatment of newly diagnosed peripheral cytomegalovirus retinitis in patients with AIDS. American Journal of Ophthalmology 133(4): 467-474, 2002.

Vitravene Study Group. Randomized dose-comparison studies of intravitreous fomivirsen for treatment of cytomegalovirus retinitis that has reactivated or is persistently active despite other therapies in patients with AIDS. American Journal of Ophthalmology 133(4): 475-483, 2002.

Vitravene Study Group. Safety of intravitreous fomivirsen of treatment of cytomegalovirus retinitis in patients with AIDS. American Journal of Ophthalmology 133(4): 484-498, 2002.

Grillone, L.R. and Lanz, R. Fomivirsen. Drugs Today (Barc). 2001 Apr;37(4):245-255.


Medicinal Chemistry/Formulations of Antisense Oligonucleotides

Tillman LG, Geary RS, Hardee GE. Oral delivery of antisense oligonucleotides in man. J Pharm Sci. 2008 Jan;97(1):225-236.

Bijsterbosch, M.K., Manoharan, M., Dorland, R., Van Veghel, R., Biessen, E.A., Van Berkel, T.J. bis-Cholesteryl-conjugated phosphorothioate oligodeoxynucleotides are highly selectively taken up by the liver. J Pharmacol Exp Ther. 302(2):619-26, 2002.

Maier, M.A., Leeds, J.M., Balow, G., Springer, R.H., Bharadwaj, R., and Manoharan, M. Nuclease resistance of oligonucleotides containing the tricyclic cytosine analogues phenoxazine and 9-(2-aminoethoxy)-phenoxazine (""G-clamp"") and origins of their nuclease resistance properties. Biochemistry. 2002 Jan 29;41(4):1323-7.

Cook, P. D. Medicinal chemistry of antisense oligonucleotides. Antisense drug technology: Principles, strategies, and applications (ed. Crooke, S. T.), pp. 29-56. Marcel Dekker, New York, 2001.

Karras J.G., Maier, M.A., Lu, T., Watt, A., Manoharan, M. Peptide Nucleic acids are potent modulators of endogenous pre-mrna splicing of the murine interleukin-5 receptor-alpha chain. Biochemistry. 2001 Jul 3;40(26):7853-9.

Manoharan, M. Oligonucleotide conjugates in antisense technology. Antisense drug technology: Principles, strategies, and applications (ed. Crooke, S. T.), pp. 391-470. Marcel Dekker, New York, 2001.

Sazani, P., Kang, S.H., Maier, M.A., Wei, C., DillmAn, J., Manoharan, M., Kole, R., Nuclear antisense effects of neutral anionic and cationic oligonucleotide analogues. Nucleic Acid Res. (29) 19: 3965-3974, 2001.

Mehta, R.C., Stecker, K.K., Cooper, S.R., Templin, M.V., Tsai, Y.J., Condon, T.P., Bennett, C.F., Hardee, G.E. Intercellular adhesion molecule-1 suppression in skin by topical delivery of anti-sense oligonucleotides. J Invest Dermatol.115(5):805-812, 2000.

Templin, M. V., Levin, A. A., Graham, M. J., Aberg, P. M., Axelsson, B. I., Butler, M., Geary, R. S., and Bennett, C. F. Pharmacokinetic and toxicity profile of a phosphorothioate oligonucleotide following inhalation delivery to lung in mice. Antisense Nucleic Acid Drug Dev. 10(5):359-368, 2000.


Antisense Oligonucleotide Manufacturing

Wang Z, Olsen P, Ravikumar VT. A Novel Universal Linker for Efficient Synthesis of Phosphorothioate Oligonucleotides. Nucleosides Nucleotides Nucleic Acids. 2007;26(3):259-69.

Krotz, A. H., Cole, D.L. , Ravikumar, V. T. Synthesis of antisense oligonucleotides with minimum depurination. Nucleosides Nucleotides Nucleic Acids. 22(2):129-34, 2003.

Pon, R.T., Yu, S., Sanghvi, Y.S. Multiple oligonucleotides synthesis in tandem on solid-phase supports for small and large scale synthesis. Nucleosides Nucleotides Nucleic Acids. 20(4-7):985-989, 2001.

Sanghvi, Y.S., Ravikumar, VT., Scozzari, A.N., Cole, D.L. Applications of green chemistry in the manufacture of oligonucleotide drugs. Pure App Chem. 73(1):175-180, 2001.


Ibis Biosciences - IBIS T5000

Hannis JC, Manalili SM, Hall TA, Ranken R, White N, Sampath R, Blyn LB, Ecker DJ, Mandrell RE, Fagerquist CK, Bates AH, Miller WG, Hofstadler SA. High resolution genotyping of campylobacter using PCR and high-throughput mass spectrometry. J Clin Microbiol. 2008 February 13;[Epub ahead of print].

Sampath R, Russell KL, Massire C, Eshoo MW, Harpin V, Blyn LB, Melton R, Ivy C, Pennella T, Li F, Levene H, Hall TA, Libby B, Fan N, Walcott DJ, Ranken R, Pear M, Schink A, Gutierrez J, Drader J, Moore D, Metzgar D, Addington L, Rothman R, Gaydos CA, Yang S, St.. George K, Fuschino ME, Dean AB, Stallknecht DE, Goekjian G, Yingst S, Monteville M, Saad MD, Whitehouse CA, Baldwin C, Rudnick KH, Hofstadler SA, Lemon SM, Ecker DJ. Global Surveillance of Emerging Influenza Virus Genotypes by Mass Spectrometry. PLoS ONE. 2007 May.

Sampath R, Hall TA, Massire C, Li F, Blyn LB, Eshoo MW, Hofstadler SA, Ecker DJ. Rapid Identification of Emerging Infectious Agents Using PCR and Electrospray Ionization Mass Spectrometry. Ann N Y Acad Sci. 2007 May;1102:109-20.

Jiang Y, Hall TA, Hofstadler SA, Naviaux RK. Mitochondrial DNA Mutation Detection by Electrospray Mass Spectrometry. Clin Chem. 2007 Feb;53(2):195-203. Epub 2006 Dec 7.

Ecker DJ, Drader JJ, Gutierrez J, Gutierrez A, Hannis JC, Schink A, Sampath R, Blyn LB, Eshoo MW, Hall TA, Tobarmosquera M, Jiang Y, Sannes-Lowery KA, Cummins LL, Libby B, Walcott DJ, Massire C, Ranken R, Manalili S, Ivy C, Melton R, Levene H, Harpin V, Li F, White N, Pear M, Ecker JA, Samant V, Knize D, Robbins D, Rudnick K, Hajjar F, Hofstadler SA. The Ibis T5000 Universal Biosensor: An Automated Platform for Pathogen Identification and Strain Typing. JALA. 2006 Dec; 6(11):341-351.

Hujer KM, Hujer AM, Hulten EA, Bajaksouzian S, Adams JM, Donskey CJ, Ecker DJ, Massire C, Eshoo MW, Sampath R, Thomson JM, Rather PN, Craft DW, Fishbain JT, Ewell AJ, Jacobs MR, Paterson DL, Bonomo RA. Analysis of antibiotic resistance genes in multidrug-resistant Acinetobacter sp. isolates from military and civilian patients treated at the Walter Reed Army Medical Center. Antimicrob Agents Chemother. 2006 Dec;50(12):4114-23..

Russell KL, Broderick MP, Franklin SE, Blyn LB, Freed NE, Moradi E, Ecker DJ, Kammerer PE, Osuna MA, Kajon AE, Morn CB, Ryan MA. Transmission dynamics and prospective environmental sampling of adenovirus in a military recruit setting. J Infect Dis. 2006 Oct 1;194(7):877-85.

Ecker JA, Massire C, Hall TA, Ranken R, Pennella TT, Agasino Ivy C, Blyn LB, Hofstadler SA, Endy TP, Scott PT, Lindler L, Hamilton T, Gaddy C, Snow K, Pe M, Fishbain J, Craft D, Deye G, Riddell S, Milstrey E, Petruccelli B, Brisse S, Harpin V, Schink A, Ecker DJ, Sampath R, Eshoo MW. Identification of Acinetobacter species and genotyping of Acinetobacter baumannii by multilocus PCR and mass spectrometry. J Clin Microbiol. 2006 Aug;44(8):2921-32.

Hofstadler SA, Sannes-Lowery KA. Applications of ESI-MS in drug discovery: interrogation of noncovalent complexes.. Nat Rev Drug Discov. 2006 Jul;5(7):585-95.

Hofstadler SA, Hari KL, Sampath R, Blyn LB, Eshoo MW, Ecker DJ, Detection of Microbial Agents Using Broad-range PRC with Detection by Mass Spectrometry: The TIGER Concept. Chapter in Encyclopedia of Rapid Microbiological Methods. 2006

Ecker DJ and Carroll KC. Investments in High-Payoff Technologies Could Reduce Toll of Infections. ASM News. 71(12): 576-581

Hofstadler SA, Drader JJ, Schink A. Selective Ion Filtering by Digital Thresholding: A Method To Unwind Complex ESI-Mass Spectra and Eliminate Signals from Low Molecular Weight Chemical Noise. Anal Chem. 78(2): 372-378. January 15, 2006

Hall TA, Budowle B, Jiang Y, Blyn L, Eshoo M, Sannes-Lowery KA, Sampath R, Drader JJ, Hannis JC, Harrell P, Samant V, White N, Ecker DJ, Hofstadler SA Base composition analysis of human mitochondrial DNA using electrospray ionization mass spectrometry: A novel tool for the identification and differentiation of humans.
Anal Biochem. 2005 Jul 26

Hofstadler S.A., Sampath, R., Blyn, L.B., Eshoo, M.W., Hall, T.A., Jiang, Y., Drader, J.J., Hannis, J.C., Sannes-Lowery, K.A., Cummins, L.L., Libby, B., Walcott, D.J., Schink, A., Massire, C., Ranken, R., Gutierrez, J., Manalili, S., Ivy, C., Melton, R., Levene, H., Barrett-Wilt, G., Li, F., Zapp, V., White, N., Samant, V., McNeil, J.A., Knize, D., Robbins, D., Rudnick, K., Desai, A., Moradi, E. and Ecker, D.J. (2005). TIGER: The Universal Biosensor.Int. J. Mass Spectrom. 242(1): 23-41

Ecker et al; Rapid identification and strain-typing of respiratory pathogens for epidemic surveillance. (2005) Proc. Natl. Acad. Sci(USA): 102(22), 8012-17

Sampath R., S.A., Hofstadler S.A., Blyn, L.B., Eshoo, M.W., Hall, T.A., Massire, C., Levene, H.M., Hannis, J.C., Harrell, P.M., Neuman, B., Buchmeier, M.J., Jiang, Y., Ranken, R., Drader, J.J., Samant, V., Griffey, R.H., McNeil, J.A., Crooke, S.T. and Ecker, D.J. (2005). Rapid Identification of Emerging Pathogens: Coronavirus. Emerg Infect Dis. 11(3):

Ecker DE, Sampath R, Willett P, Wyatt JR, Samant V, Massire C, Hall TA, Hari KL, McNeil JA, Buchen-Osmond C, Budowle B. The Microbial Rosetta Stone Database: A compilation of global and emerging infectious microorganisms and bioterrorist threat agents. BMC Microbiology 2005, 5:19.

Ecker DJ, Sampath R, Willett P, Samant V, Massire C, Hall TA, Hari K, McNeil JA, Buchen-Osmond C, Budowle B. The Microbial Rosetta Stone Database: A common structure for microbial biosecurity threat agents. J Forensic Sci.2005 Nov;50(6):1380-5.

Van Ert M.N., Hofstadler, S.A., Jiang, Y., Busch, J.D., Wagner, D.M., Drader, J.J., Ecker, D.J., Hannis, J.C., Huynh, L.Y., Schupp, J.M., Simonson, T.S. and Keim, P. (2004). Mass spectrometry provides accurate characterization of two genetic marker types in Bacillus anthracis. Biotechniques. 37(4): 642-4, 646, 648.

Sampath, R. & Ecker, D. J. in Forum on Microbial Threats: Learning from SARS: Preparing for the Next Disease Outbreak -- Workshop Summary (ed. Knobler, S. E., Mahmoud, A., Lemon, S.) 181-185 (The National Academies Press, Washington, D.C., 2004).

Jiang, Y. & Hofstadler, S. A. A highly efficient and automated method of purifying and desalting PCR products for analysis by electrospray ionization mass spectrometry. Anal. Biochem.316 , 50-57 (2003).