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Items: 1 to 50 of 201

1.

Targeting Translation Termination Machinery with Antisense Oligonucleotides for Diseases Caused by Nonsense Mutations.

Huang L, Aghajan M, Quesenberry T, Low A, Murray SF, Monia BP, Guo S.

Nucleic Acid Ther. 2019 May 9. doi: 10.1089/nat.2019.0779. [Epub ahead of print]

PMID:
31070517
2.

Thrombin signaling promotes pancreatic adenocarcinoma through PAR-1-dependent immune evasion.

Yang Y, Stang A, Schweickert PG, Lanman NA, Paul EN, Monia BP, Revenko AS, Palumbo JS, Mullins ES, Elzey BD, Janssen EM, Konieczny SF, Flick MJ.

Cancer Res. 2019 May 2. pii: canres.3206.2018. doi: 10.1158/0008-5472.CAN-18-3206. [Epub ahead of print]

PMID:
31048498
3.

Nonsense Mediated RNA Decay Pathway Inhibition Restores Expression and Function of W1282X CFTR.

Keenan MM, Huang L, Jordan NJ, Wong E, Cheng Y, Valley HC, Mahiou J, Liang F, Bihler H, Mense M, Guo S, Monia BP.

Am J Respir Cell Mol Biol. 2019 Mar 5. doi: 10.1165/rcmb.2018-0316OC. [Epub ahead of print]

PMID:
30836009
4.

IONIS-PKKRx a Novel Antisense Inhibitor of Prekallikrein and Bradykinin Production.

Ferrone JD, Bhattacharjee G, Revenko AS, Zanardi TA, Warren MS, Derosier FJ, Viney NJ, Pham NC, Kaeser GE, Baker BF, Schneider E, Hughes SG, Monia BP, MacLeod AR.

Nucleic Acid Ther. 2019 Apr;29(2):82-91. doi: 10.1089/nat.2018.0754. Epub 2019 Feb 28.

5.

Inotersen (transthyretin-specific antisense oligonucleotide) for treatment of transthyretin amyloidosis.

Benson MD, Dasgupta NR, Monia BP.

Neurodegener Dis Manag. 2019 Feb;9(1):25-30. doi: 10.2217/nmt-2018-0037. Epub 2018 Dec 18.

PMID:
30561247
6.

STAT3 antisense oligonucleotide AZD9150 in a subset of patients with heavily pretreated lymphoma: results of a phase 1b trial.

Reilley MJ, McCoon P, Cook C, Lyne P, Kurzrock R, Kim Y, Woessner R, Younes A, Nemunaitis J, Fowler N, Curran M, Liu Q, Zhou T, Schmidt J, Jo M, Lee SJ, Yamashita M, Hughes SG, Fayad L, Piha-Paul S, Nadella MVP, Xiao X, Hsu J, Revenko A, Monia BP, MacLeod AR, Hong DS.

J Immunother Cancer. 2018 Nov 16;6(1):119. doi: 10.1186/s40425-018-0436-5.

7.

Targeted delivery of antisense oligonucleotides to pancreatic β-cells.

Ämmälä C, Drury WJ 3rd, Knerr L, Ahlstedt I, Stillemark-Billton P, Wennberg-Huldt C, Andersson EM, Valeur E, Jansson-Löfmark R, Janzén D, Sundström L, Meuller J, Claesson J, Andersson P, Johansson C, Lee RG, Prakash TP, Seth PP, Monia BP, Andersson S.

Sci Adv. 2018 Oct 17;4(10):eaat3386. doi: 10.1126/sciadv.aat3386. eCollection 2018 Oct.

8.

Reducing dynamin 2 (DNM2) rescues DNM2-related dominant centronuclear myopathy.

Buono S, Ross JA, Tasfaout H, Levy Y, Kretz C, Tayefeh L, Matson J, Guo S, Kessler P, Monia BP, Bitoun M, Ochala J, Laporte J, Cowling BS.

Proc Natl Acad Sci U S A. 2018 Oct 23;115(43):11066-11071. doi: 10.1073/pnas.1808170115. Epub 2018 Oct 5.

9.

Hereditary transthyretin amyloidosis: baseline characteristics of patients in the NEURO-TTR trial.

Waddington-Cruz M, Ackermann EJ, Polydefkis M, Heitner SB, Dyck PJ, Barroso FA, Wang AK, Berk JL, Dyck PJB, Monia BP, Hughes SG, Tai L, Jesse Kwoh T, Jung SW, Coelho T, Benson MD, Gertz MA.

Amyloid. 2018 Sep;25(3):180-188. doi: 10.1080/13506129.2018.1503593. Epub 2018 Aug 31.

PMID:
30169969
10.

Antisense oligonucleotide targeting of mRNAs encoding ENaC subunits α, β, and γ improves cystic fibrosis-like disease in mice.

Zhao C, Crosby J, Lv T, Bai D, Monia BP, Guo S.

J Cyst Fibros. 2019 May;18(3):334-341. doi: 10.1016/j.jcf.2018.07.006. Epub 2018 Aug 10.

PMID:
30100257
11.

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.

Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH 3rd, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceição I, Schmidt HH, Vita G, Campistol JM, Gamez J, Gorevic PD, Gane E, Shah AM, Solomon SD, Monia BP, Hughes SG, Kwoh TJ, McEvoy BW, Jung SW, Baker BF, Ackermann EJ, Gertz MA, Coelho T.

N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793.

12.

Antisense suppression of the nonsense mediated decay factor Upf3b as a potential treatment for diseases caused by nonsense mutations.

Huang L, Low A, Damle SS, Keenan MM, Kuntz S, Murray SF, Monia BP, Guo S.

Genome Biol. 2018 Jan 15;19(1):4. doi: 10.1186/s13059-017-1386-9.

13.

Reduction in ocular complement factor B protein in mice and monkeys by systemic administration of factor B antisense oligonucleotide.

Grossman TR, Carrer M, Shen L, Johnson RB, Hettrick LA, Henry SP, Monia BP, McCaleb ML.

Mol Vis. 2017 Aug 10;23:561-571. eCollection 2017.

14.

Knockdown of Z Mutant Alpha-1 Antitrypsin In Vivo Using Modified DNA Antisense Oligonucleotides.

Aghajan M, Guo S, Monia BP.

Methods Mol Biol. 2017;1639:127-138. doi: 10.1007/978-1-4939-7163-3_12.

PMID:
28752452
15.

Navigating the Future of Cardiovascular Drug Development-Leveraging Novel Approaches to Drive Innovation and Drug Discovery: Summary of Findings from the Novel Cardiovascular Therapeutics Conference.

Povsic TJ, Scott R, Mahaffey KW, Blaustein R, Edelberg JM, Lefkowitz MP, Solomon SD, Fox JC, Healy KE, Khakoo AY, Losordo DW, Malik FI, Monia BP, Montgomery RL, Riesmeyer J, Schwartz GG, Zelenkofske SL, Wu JC, Wasserman SM, Roe MT.

Cardiovasc Drugs Ther. 2017 Aug;31(4):445-458. doi: 10.1007/s10557-017-6739-9. Review.

PMID:
28735360
16.

Targeting KRAS-dependent tumors with AZD4785, a high-affinity therapeutic antisense oligonucleotide inhibitor of KRAS.

Ross SJ, Revenko AS, Hanson LL, Ellston R, Staniszewska A, Whalley N, Pandey SK, Revill M, Rooney C, Buckett LK, Klein SK, Hudson K, Monia BP, Zinda M, Blakey DC, Lyne PD, Macleod AR.

Sci Transl Med. 2017 Jun 14;9(394). pii: eaal5253. doi: 10.1126/scitranslmed.aal5253. Erratum in: Sci Transl Med. 2017 Jul 26;9(400):.

PMID:
28615361
17.

Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice.

Tasfaout H, Buono S, Guo S, Kretz C, Messaddeq N, Booten S, Greenlee S, Monia BP, Cowling BS, Laporte J.

Nat Commun. 2017 Jun 7;8:15661. doi: 10.1038/ncomms15661.

18.

Inhaled ENaC antisense oligonucleotide ameliorates cystic fibrosis-like lung disease in mice.

Crosby JR, Zhao C, Jiang C, Bai D, Katz M, Greenlee S, Kawabe H, McCaleb M, Rotin D, Guo S, Monia BP.

J Cyst Fibros. 2017 Nov;16(6):671-680. doi: 10.1016/j.jcf.2017.05.003. Epub 2017 May 20.

PMID:
28539224
19.

Treatment of transthyretin cardiomyopathy with a TTR-specific antisense oligonucleotide (IONIS-TTRRx).

Benson MD, Ackermann EJ, Monia BP.

Amyloid. 2017 Mar;24(sup1):134-135. doi: 10.1080/13506129.2017.1280015. No abstract available.

PMID:
28434337
20.

Use of antisense oligonucleotides to correct the splicing error in ISCU myopathy patient cell lines.

Holmes-Hampton GP, Crooks DR, Haller RG, Guo S, Freier SM, Monia BP, Rouault TA.

Hum Mol Genet. 2016 Dec 1;25(23):5178-5187. doi: 10.1093/hmg/ddw338.

21.

Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides.

Ackermann EJ, Guo S, Benson MD, Booten S, Freier S, Hughes SG, Kim TW, Jesse Kwoh T, Matson J, Norris D, Yu R, Watt A, Monia BP.

Amyloid. 2016 Sep;23(3):148-157. Epub 2016 Jun 29.

PMID:
27355239
22.

Evaluation of Therapeutic Oligonucleotides for Familial Amyloid Polyneuropathy in Patient-Derived Hepatocyte-Like Cells.

Niemietz CJ, Sauer V, Stella J, Fleischhauer L, Chandhok G, Guttmann S, Avsar Y, Guo S, Ackermann EJ, Gollob J, Monia BP, Zibert A, Schmidt HH.

PLoS One. 2016 Sep 1;11(9):e0161455. doi: 10.1371/journal.pone.0161455. eCollection 2016.

23.

AZD9150, a next-generation antisense oligonucleotide inhibitor of STAT3 with early evidence of clinical activity in lymphoma and lung cancer.

Hong D, Kurzrock R, Kim Y, Woessner R, Younes A, Nemunaitis J, Fowler N, Zhou T, Schmidt J, Jo M, Lee SJ, Yamashita M, Hughes SG, Fayad L, Piha-Paul S, Nadella MV, Mohseni M, Lawson D, Reimer C, Blakey DC, Xiao X, Hsu J, Revenko A, Monia BP, MacLeod AR.

Sci Transl Med. 2015 Nov 18;7(314):314ra185. doi: 10.1126/scitranslmed.aac5272.

24.

Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration.

Murray SF, Jazayeri A, Matthes MT, Yasumura D, Yang H, Peralta R, Watt A, Freier S, Hung G, Adamson PS, Guo S, Monia BP, LaVail MM, McCaleb ML.

Invest Ophthalmol Vis Sci. 2015 Oct;56(11):6362-75. doi: 10.1167/iovs.15-16400.

25.

Combination of Tmprss6- ASO and the iron chelator deferiprone improves erythropoiesis and reduces iron overload in a mouse model of beta-thalassemia intermedia.

Casu C, Aghajan M, Oikonomidou PR, Guo S, Monia BP, Rivella S.

Haematologica. 2016 Jan;101(1):e8-e11. doi: 10.3324/haematol.2015.133348. Epub 2015 Sep 24. No abstract available.

26.

Reversing Antisense Oligonucleotide Activity with a Sense Oligonucleotide Antidote: Proof of Concept Targeting Prothrombin.

Crosby JR, Zhao C, Zhang H, MacLeod AR, Guo S, Monia BP.

Nucleic Acid Ther. 2015 Dec;25(6):297-305. doi: 10.1089/nat.2015.0560. Epub 2015 Sep 21.

PMID:
26390010
27.

Inhibition of the alternative complement pathway by antisense oligonucleotides targeting complement factor B improves lupus nephritis in mice.

Grossman TR, Hettrick LA, Johnson RB, Hung G, Peralta R, Watt A, Henry SP, Adamson P, Monia BP, McCaleb ML.

Immunobiology. 2016 Jun;221(6):701-8. doi: 10.1016/j.imbio.2015.08.001. Epub 2015 Aug 10.

28.

Genetic diminution of circulating prothrombin ameliorates multiorgan pathologies in sickle cell disease mice.

Arumugam PI, Mullins ES, Shanmukhappa SK, Monia BP, Loberg A, Shaw MA, Rizvi T, Wansapura J, Degen JL, Malik P.

Blood. 2015 Oct 8;126(15):1844-55. doi: 10.1182/blood-2015-01-625707. Epub 2015 Aug 18.

29.

Colon Cancer Growth and Dissemination Relies upon Thrombin, Stromal PAR-1, and Fibrinogen.

Adams GN, Rosenfeldt L, Frederick M, Miller W, Waltz D, Kombrinck K, McElhinney KE, Flick MJ, Monia BP, Revenko AS, Palumbo JS.

Cancer Res. 2015 Oct 1;75(19):4235-43. doi: 10.1158/0008-5472.CAN-15-0964. Epub 2015 Aug 3.

30.

Partial Hepatectomy Induced Long Noncoding RNA Inhibits Hepatocyte Proliferation during Liver Regeneration.

Huang L, Damle SS, Booten S, Singh P, Sabripour M, Hsu J, Jo M, Katz M, Watt A, Hart CE, Freier SM, Monia BP, Guo S.

PLoS One. 2015 Jul 24;10(7):e0132798. doi: 10.1371/journal.pone.0132798. eCollection 2015.

31.

Arterial thrombosis is accelerated in mice deficient in histidine-rich glycoprotein.

Vu TT, Zhou J, Leslie BA, Stafford AR, Fredenburgh JC, Ni R, Qiao S, Vaezzadeh N, Jahnen-Dechent W, Monia BP, Gross PL, Weitz JI.

Blood. 2015 Apr 23;125(17):2712-9. doi: 10.1182/blood-2014-11-611319. Epub 2015 Feb 17.

32.

Generation 2.5 antisense oligonucleotides targeting the androgen receptor and its splice variants suppress enzalutamide-resistant prostate cancer cell growth.

Yamamoto Y, Loriot Y, Beraldi E, Zhang F, Wyatt AW, Al Nakouzi N, Mo F, Zhou T, Kim Y, Monia BP, MacLeod AR, Fazli L, Wang Y, Collins CC, Zoubeidi A, Gleave M.

Clin Cancer Res. 2015 Apr 1;21(7):1675-87. doi: 10.1158/1078-0432.CCR-14-1108. Epub 2015 Jan 29.

33.

Transthyretin Antisense Oligonucleotides Lower Circulating RBP4 Levels and Improve Insulin Sensitivity in Obese Mice.

Zemany L, Bhanot S, Peroni OD, Murray SF, Moraes-Vieira PM, Castoldi A, Manchem P, Guo S, Monia BP, Kahn BB.

Diabetes. 2015 May;64(5):1603-14. doi: 10.2337/db14-0970. Epub 2014 Dec 18.

34.

Factor XI antisense oligonucleotide for prevention of venous thrombosis.

Büller HR, Bethune C, Bhanot S, Gailani D, Monia BP, Raskob GE, Segers A, Verhamme P, Weitz JI; FXI-ASO TKA Investigators.

N Engl J Med. 2015 Jan 15;372(3):232-40. doi: 10.1056/NEJMoa1405760. Epub 2014 Dec 7.

35.

Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease.

Guo S, Booten SL, Watt A, Alvarado L, Freier SM, Teckman JH, McCaleb ML, Monia BP.

Rare Dis. 2014 Mar 12;2:e28511. doi: 10.4161/rdis.28511. eCollection 2014.

36.

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice.

Prakash TP, Graham MJ, Yu J, Carty R, Low A, Chappell A, Schmidt K, Zhao C, Aghajan M, Murray HF, Riney S, Booten SL, Murray SF, Gaus H, Crosby J, Lima WF, Guo S, Monia BP, Swayze EE, Seth PP.

Nucleic Acids Res. 2014 Jul;42(13):8796-807. doi: 10.1093/nar/gku531. Epub 2014 Jul 3.

37.

Factor XI regulates pathological thrombus formation on acutely ruptured atherosclerotic plaques.

van Montfoort ML, Kuijpers MJ, Knaup VL, Bhanot S, Monia BP, Roelofs JJ, Heemskerk JW, Meijers JC.

Arterioscler Thromb Vasc Biol. 2014 Aug;34(8):1668-73. doi: 10.1161/ATVBAHA.114.303209. Epub 2014 Jun 19.

PMID:
24947525
38.

Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy.

Lieberman AP, Yu Z, Murray S, Peralta R, Low A, Guo S, Yu XX, Cortes CJ, Bennett CF, Monia BP, La Spada AR, Hung G.

Cell Rep. 2014 May 8;7(3):774-84. doi: 10.1016/j.celrep.2014.02.008. Epub 2014 Apr 16.

39.

Nicotinamide N-methyltransferase knockdown protects against diet-induced obesity.

Kraus D, Yang Q, Kong D, Banks AS, Zhang L, Rodgers JT, Pirinen E, Pulinilkunnil TC, Gong F, Wang YC, Cen Y, Sauve AA, Asara JM, Peroni OD, Monia BP, Bhanot S, Alhonen L, Puigserver P, Kahn BB.

Nature. 2014 Apr 10;508(7495):258-62. doi: 10.1038/nature13198.

40.

Selective depletion of factor XI or factor XII with antisense oligonucleotides attenuates catheter thrombosis in rabbits.

Yau JW, Liao P, Fredenburgh JC, Stafford AR, Revenko AS, Monia BP, Weitz JI.

Blood. 2014 Mar 27;123(13):2102-7. doi: 10.1182/blood-2013-12-540872. Epub 2014 Feb 5.

41.

Antisense oligonucleotide treatment ameliorates alpha-1 antitrypsin-related liver disease in mice.

Guo S, Booten SL, Aghajan M, Hung G, Zhao C, Blomenkamp K, Gattis D, Watt A, Freier SM, Teckman JH, McCaleb ML, Monia BP.

J Clin Invest. 2014 Jan;124(1):251-61. doi: 10.1172/JCI67968. Epub 2013 Dec 20.

42.

9th annual meeting of the Oligonucleotide Therapeutics Society Naples, Italy | October 6-8, 2013. Session summaries.

Monia BP; Board Of The Oligonucleotide Therapeutics Society.

Nucleic Acid Ther. 2013 Dec;23(6):450-8. doi: 10.1089/nat.2013.1505. No abstract available.

PMID:
24329415
43.

Identification of REST-regulated genes and pathways using a REST-targeted antisense approach.

Sedaghat Y, Bui HH, Mazur C, Monia BP.

Nucleic Acid Ther. 2013 Dec;23(6):389-400. doi: 10.1089/nat.2013.0445.

PMID:
24329414
44.

Characterization of target mRNA reduction through in situ RNA hybridization in multiple organ systems following systemic antisense treatment in animals.

Hung G, Xiao X, Peralta R, Bhattacharjee G, Murray S, Norris D, Guo S, Monia BP.

Nucleic Acid Ther. 2013 Dec;23(6):369-78. doi: 10.1089/nat.2013.0443. Epub 2013 Oct 26. Erratum in: Nucleic Acid Ther. 2014 Apr;24(2):178.

PMID:
24161045
45.

Peripheral reduction of FGFR4 with antisense oligonucleotides increases metabolic rate and lowers adiposity in diet-induced obese mice.

Yu XX, Watts LM, Manchem VP, Chakravarty K, Monia BP, McCaleb ML, Bhanot S.

PLoS One. 2013 Jul 29;8(7):e66923. doi: 10.1371/journal.pone.0066923. Print 2013.

46.

Preclinical evaluation of the toxicological effects of a novel constrained ethyl modified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys.

Burel SA, Han SR, Lee HS, Norris DA, Lee BS, Machemer T, Park SY, Zhou T, He G, Kim Y, MacLeod AR, Monia BP, Lio S, Kim TW, Henry SP.

Nucleic Acid Ther. 2013 Jun;23(3):213-27. doi: 10.1089/nat.2013.0422.

PMID:
23692080
47.

Inhibition of vascular permeability by antisense-mediated inhibition of plasma kallikrein and coagulation factor 12.

Bhattacharjee G, Revenko AS, Crosby JR, May C, Gao D, Zhao C, Monia BP, MacLeod AR.

Nucleic Acid Ther. 2013 Jun;23(3):175-87. doi: 10.1089/nat.2013.0417. Epub 2013 Apr 12.

PMID:
23582057
48.

Antithrombotic effect of antisense factor XI oligonucleotide treatment in primates.

Crosby JR, Marzec U, Revenko AS, Zhao C, Gao D, Matafonov A, Gailani D, MacLeod AR, Tucker EI, Gruber A, Hanson SR, Monia BP.

Arterioscler Thromb Vasc Biol. 2013 Jul;33(7):1670-8. doi: 10.1161/ATVBAHA.113.301282. Epub 2013 Apr 4. Erratum in: Arterioscler Thromb Vasc Biol. 2013 Aug;33(8):e127. Arterioscler Thromb Vasc Biol. 2013 Oct;33(10):e130.

49.

Reducing TMPRSS6 ameliorates hemochromatosis and β-thalassemia in mice.

Guo S, Casu C, Gardenghi S, Booten S, Aghajan M, Peralta R, Watt A, Freier S, Monia BP, Rivella S.

J Clin Invest. 2013 Apr;123(4):1531-41. doi: 10.1172/JCI66969. Epub 2013 Mar 25.

50.

Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy.

Ackermann EJ, Guo S, Booten S, Alvarado L, Benson M, Hughes S, Monia BP.

Amyloid. 2012 Jun;19 Suppl 1:43-4. doi: 10.3109/13506129.2012.673140. Epub 2012 Apr 12.

PMID:
22494066

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