Founder's Site

James E. Summerton, Ph.D.

[6] Morpholino Oligos pre-clinical and clinical papers

[ link 6 ]

Morpholino oligos
for treatment of diseases

(Representative citations, with hundreds of others at:


Breast cancer

Antitumorigenic potential of STAT3 alternative splicing modulation.
Zammarchi F, de Stanchina E, Bournazou E, Supakorndej T, Martires K, Riedel E, Corben AD, Bromberg JF, Cartegni L. Proc Natl Acad Sci U S A. 2011 October 25; 108(43): 17779–17784. Published online 2011 October 17. doi: 10.1073/pnas.1108482108.

Nitric Oxide Synthase and Breast Cancer: Role of TIMP-1 in NO-mediated Akt Activation.
Ridnour LA, Barasch KM, Windhausen AN, Dorsey TH, Lizardo MM, Yfantis HG, Lee DH, Switzer CH, Cheng RYS, Heinecke JL, Brueggmann E, Hines HB, Khanna C, Glynn SA, Ambs S, Wink DA. PLoS ONE. 2012;7(9): e44081. doi:10.1371/journal.pone.0044081.

Colon cancer

Morpholino-Mediated Isoform Modulation of Vascular Endothelial Growth Factor Receptor-2 (VEGFR2) Reduces Colon Cancer Xenograft Growth.
Stagg BC, Uehara H, Lambert N, Rai R, Gupta I, Radmall B, Bates T, Ambati BK. Cancers. 2014;6(4):2330-42. doi:10.3390/cancers6042330.


Inhibition of laminin-8 in vivo using a novel poly(malic acid)-based carrier reduces glioma angiogenesis.
Fujita M, Khazenzon NM, Ljubimov AV, Lee BS, Virtanen I, Holler E, Black KL, Ljubimova JY. Angiogenesis. 2006;9(4):183-91. Epub 2006 Nov 16.

Hepatocellular carcinoma

Targeted inhibition of tumor-specific glutaminase diminishes cell-autonomous tumorigenesis.
Xiang Y, Stine ZE, Xia J, Lu Y, O’Connor RS, Altman BJ, Hsieh AL, Gouw AM, Thomas AG, Gao P, Sun L, Song L, Yan B, Slusher BS, Zhuo J, Ooi LL, Lee CGL, Mancuso A, McCallion AS, Le A, Milone MC, Rayport S, Felsher DW, Dang CV. J Clin Invest. 2015;[Epub ahead of print] doi:10.1172/JCI75836.

Blockade of CD47 mediated CTSS-PAR2 signaling provides a therapeutic target for hepatocellular carcinoma.
Lee TKW, Cheung VCH, Lu P, Lau EYT, Ma S, Tang KH, Tong M, Lo J, Ng IOL. Hepatology. 2014;[Epub ahead of print] doi:10.1002/hep.27070.

Lung cancer

E2F8 as a Novel Therapeutic Target for Lung Cancer.
Park SA, Platt J, Lee JW, López-Giráldez F, Herbst RS, Koo JS. J Natl Cancer Inst. 2015 Jun 18;107(9). pii: djv151. doi: 10.1093/jnci/djv151. Print 2015 Sep.

Lymphoma & Leukemia

Two-Step Pretargeted Nanotherapy for CD20 Crosslinking May Achieve Superior Anti-Lymphoma Efficacy to Rituximab.
Chu TW, Zhang R, Yang J, Chao MP, Shami PJ, Kopeček J. A Theranostics. 2015;5(8):834-46. doi:10.7150/thno.12040.

Drug-Free Macromolecular Therapeutics Induce Apoptosis of Patient Chronic Lymphocytic Leukemia Cells.
Chu TW, Kosak KM, Shami PJ, Kopeček J. Drug Deliv Transl Res. 2014 Dec;4(5-6):389-394.

Therapeutic antagonists of microRNAs deplete leukemia-initiating cell activity.
Velu CS, Chaubey A, Phelan JD, Horman SR, Wunderlich M, Guzman ML, Jegga AG, Zeleznik-Le NJ, Chen J, Mulloy JC, Cancelas JA, Jordan CT, Aronow BJ, Marcucci G, Bhat B, Gebelein B, Grimes HL. J Clin Invest. 2014 Jan 2;124(1):222-36. doi: 10.1172/JCI66005. Epub 2013 Dec 16.

Nijmegen Breakage Syndrome

Directed Alternative Splicing in Nijmegen Breakage Syndrome: Proof of Principle Concerning Its Therapeutical Application.
Salewsky B, Hildebrand G, Rothe S, Parplys AC, Radszewski J, Kieslich M, Wessendorf P, Krenzlin H, Borgmann K, Nussenzweig A, Sperling K, Digweed M. Mol Ther. 2015 Aug 12. doi: 10.1038/mt.2015.144. [Epub ahead of print]

Ovarian cancer

Reversing paclitaxel resistance in ovarian cancer cells via inhibition of the ABCB1 expressing side population.
Eyre R, Harvey I, Stemke-Hale K, Lennard TW, Tyson-Capper A, Meeson AP.  Tumour Biol. 2014 Jul 4. [Epub ahead of print].

Prostate cancer

A novel antisense inhibitor of MMP-9 attenuates angiogenesis, human prostate cancer cell invasion and tumorigenicity.
London CA, Sekhon HS, Arora V, Stein DA, Iversen PL, Devi GR. Cancer Gene Ther. 2003 Nov;10(11):823-32.

Potentiation of radiation therapy

Nitric Oxide Synthase Inhibition Modulates Immune Polarization and Improves Radiation-Induced Tumor Growth Delay.
Ridnour LA, Cheng RY, Weiss JM, Soto-Pantoja DR, Basudhar D, Heinecke JL, Stewart A, DeGraff WG, Sowers A, Thetford A, Kesarwala AH, Roberts DD, Young HA, Mitchell JB, Trinchieri G, Wiltrout RH, Wink DA. Cancer Res. 2015 May 19. pii: canres.3011.2014. [Epub ahead of print].

CD47 in the Tumor Microenvironment Limits Cooperation between Antitumor T-cell Immunity and Radiotherapy.
Soto-Pantoja DR, Terabe M, Ghosh A, Ridnour LA, DeGraff WG, Wink DA, Berzofsky JA, Roberts DD. Cancer Res. 2014 Dec 1;74(23):6771-83. doi: 10.1158/0008-5472.CAN-14-0037-T. Epub 2014 Oct 8.

Genetic Diseases

Amyotrophic lateral sclerosis

EPHA4 is a disease modifier of amyotrophic lateral sclerosis in animal models and in humans.
Van Hoecke A, Schoonaert L, Lemmens R, Timmers M, Staats KA, Laird AS, Peeters E, Philips T, Goris A, Dubois B, Andersen PM, Al-Chalabi A, Thijs V, Turnley AM, van Vught PW, Veldink JH, Hardiman O, Van Den Bosch L, Gonzalez-Perez P, Van Damme P, Brown RH Jr, van den Berg LH, Robberecht W. Nat Med. 2012 Aug 26. doi: 10.1038/nm.2901. [Epub ahead of print].


Functional characterization and targeted correction of ATM mutations identified in Japanese patients with ataxia-telangiectasia.
Nakamura K, Du L, Tunuguntla R, Fike F, Cavalieri S, Morio T, Mizutani S, Brusco A, Gatti RA. Hum Mutat. 2012 Jan;33(1):198-208. doi: 10.1002/humu.21632. Epub 2011 Nov 9.

Duchenne Muscular Dystrophy

Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for the Treatment of Duchenne Muscular Dystrophy (DMD): 168 Week Update on Six-Minute Walk Test (6MWT), Pulmonary Function Testing (PFT), and Safety.
Mendell JR, Rodino-Klapac L, Sahenk Z, Rouch K, Bird L, Lowes L, Alfano L, Berry K, Lewis S, Shontz K, Flanigan K, Shilling C, Duda P, Saoud J. Oral Abstract, American Society for Gene and Cell Therapy conference. 2015.

A Phase I/IIa Clinical Trial in Duchenne Muscular Dystrophy Using Systemically Delivered Morpholino Antisense Oligomer to Skip Exon 53 (SKIP-NMD).
No Authors Listed. Human Gene Therapy Clinical Development. June 2015, 26(2): 92-95. doi:10.1089/humc.2015.2528.

Huntington’s disease

Phosphorodiamidate Morpholino Oligomers (PMOs) suppress mutant huntingtin expression and attenuate neurotoxicity.
Sun X, Marque LO, Cordner Z, Pruitt JL, Bhat M, Li P, Kannan G, Ladenheim EE, Moran TH, Margolis RL, Rudnicki DD. Hum Mol Genet. 2014;[Epub ahead of print] doi:10.1093/hmg/ddu349.

Hutchinson-Gilford Progeria

Splicing-Directed Therapy in a New Mouse Model of Human Accelerated Aging.
Osorio FG, Navarro CL, Cadiñanos J, López-Mejía IC, Quirós PM, Bartoli C, Rivera J, Tazi J, Guzmán G, Varela I, Depetris D, de Carlos F, Cobo J, Andrés V, Sandre-Giovannoli AD, Freije JM, Lévy N, López-Otín C. Sci. Transl. Med. 2011;3:106ra107 doi:10.1126/scitranslmed.3002847.

Reversal of the cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome.
Scaffidi P, Misteli T. Nat Med. 2005 Apr;11(4):440-5. Epub 2005 Mar 6.

Menkes disease

In vivo correction of a Menkes disease model using antisense oligonucleotides.
Madsen EC, Morcos PA, Mendelsohn BA, Gitlin JD. Proc Natl Acad Sci U S A. 2008 Mar 11;105(10):3909-14. Epub 2008 Mar 3.

Myotonic Dystrophy

Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy.
Wheeler TM, Lueck JD, Swanson MS, Dirksen RT, Thornton CA. J Clin Invest. 2007 Nov 15; [Epub ahead of print].

Targeting nuclear RNA for in vivo correction of myotonic dystrophy.
Wheeler T, Leger AJ, Pandey SK, MacLeod AR, Nakamori M, Cheng SH, Wentworth BM, Bennett CF, Thornton CA. Nature. 2012;488:111-5 doi:10.1038/nature11362.

Neurofibromatosis type 1

Antisense therapeutics for neurofibromatosis type 1 caused by deep intronic mutations.
Pros E, Fernández-Rodríguez J, Canet B, Benito L, Sánchez A, Benavides A, Ramos FJ, López-Ariztegui MA, Capellá G, Blanco I, Serra E, Lázaro C. Hum Mutat. 2009 Feb 24;30(3):454-462. [Epub ahead of print].

Neurofibromatosis type 2

In vitro antisense therapeutics for a deep intronic mutation causing Neurofibromatosis type 2.
Castellanos EC, Rosas I, Solanes A, Bielsa I, Lazaro C, Carrato C, Hostalot C, Prades P, Roca-Ribas F, Blanco I, Serra E. Eur J Hum Genet. 2012;[Epub ahead of print] doi:10.1038/ejhg.2012.261.

Niemann-Pick type C disease

Antisense oligonucleotide treatment for a pseudoexon-generating mutation in the NPC1 gene causing Niemann-Pick type C disease.
Rodríguez-Pascau L, Coll MJ, Vilageliu L, Grinberg D. Hum Mutat. 2009 Aug 30. [Epub ahead of print].

Polycystic kidney disease

Modulation of the Secretory Pathway Rescues Zebrafish Polycystic Kidney Disease Pathology.
Le Corre S, Eyre D, Drummond IA. J Am Soc Nephrol. 2014 Mar 13. [Epub ahead of print].

Inhibition of the P2X7 Receptor Reduces Cystogenesis in PKD.
Chang MY, Lu JK, Tian YC, Chen YC, Hung CC, Huang YH, Chen YH, Wu MS, Yang CW, Cheng YC. J Am Soc Nephrol. 2011 Jun 2. [Epub ahead of print].

Pompe Disease

Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease.
Clayton NP, Nelson CA, Weeden T, Taylor KM, Moreland RJ, Scheule RK, Phillips L, Leger AJ, Cheng SH, Wentworth BM. Mol Ther Nucleic Acids. 2014 Oct 28;3:e206. doi: 10.1038/mtna.2014.57.

Primary immunodeficiency diseases

Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases.
Du L, Gatti RA. J Immunol Methods. 2010 Dec 10. [Epub ahead of print].

Propionic and Methylmalonic Acidemia

Propionic and Methylmalonic Acidemia: Antisense Therapeutics for Intronic Variations Causing Aberrantly Spliced Messenger RNA.
Ugarte M, Aguado C, Desviat LR, Sanchez-Alcudia R, Rincon A, Perez B. Am J Hum Genet. 2007 Oct 26;81(6) [Epub ahead of print].

Spinal Muscular Atrophy

Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches.
Nizzardo M, Simone C, Dametti S, Salani S, Ulzi G, Pagliarani S, Rizzo F, Frattini E, Pagani F, Bresolin N, Comi G, Corti S. Sci Rep. 2015 Jun 30;5:11746. doi: 10.1038/srep11746.

Effect of Combined Systemic and Local Morpholino Treatment on the Spinal Muscular Atrophy Δ7 Mouse Model Phenotype.
Nizzardo N, Simone C, Salani S, Ruepp M-D, Rizzo F, Ruggieri M, Zanetta C, Brajkovic S, Moulton HM, Müehlemann O, Bresolin N, Comi GP, Corti S. Clin Therap. 2014. 36(3):340-56. doi:10.1016/j.clinthera.2014.02.004.

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in the mouse.
Porensky PM, Mitrpant C, McGovern VL, Bevan AK, Foust KD, Kaspar BK, Wilton SD, Burghes AHM. Hum Mol Genet. 2012 Apr 1;21(7):1625-38. Epub 2011 Dec 20. doi:10.1093/hmg/ddr600.

Degenerative, injury and environmental diseases


HS3ST2 expression is critical for the abnormal phosphorylation of tau in Alzheimer’s disease-related tau pathology.
Sepulveda-Diaz JE, Naini SMA, Huynh MB, Ouidja MO, Yanicostas C, Chantepie S, Villares J, Lamari F, Jospin E, van Kuppevelt TH, Mensah-Nyagan AG, Raisman-Vozari R, Soussi-Yanicostas N, Papy-Garcia D. Brain. 2015;[Epub ahead of print] doi:10.1093/brain/awv056.


Increasing survival of ischemic tissue by targeting CD47.
Isenberg JS, Romeo MJ, Abu-Asab M, Tsokos M, Oldenborg A, Pappan L, Wink DA, Frazier WA, Roberts DD. Circ. Res. 100:712-720, 2007.


Disruption of KATP channel expression in skeletal muscle by targeted oligonucleotide delivery promotes activity-linked thermogenesis.
Koganti SR, Zhu Z, Subbotina E, Gao Z, Sierra A, Proenza M, Yang L, Alekseev A, Hodgson-Zingman D, Zingman L. Mol Ther. 2015 Feb 4. doi: 10.1038/mt.2015.21. [Epub ahead of print].

Downregulation of max dimerization protein 3 is involved in decreased visceral adipose tissue by inhibiting adipocyte differentiation in zebrafish and mice.
Shimada Y, Kuroyanagi J, Zhang B, Ariyoshi M, Umemoto N, Nishimura Y, Tanaka T. Int J Obes (Lond). 2013 Nov 20. doi: 10.1038/ijo.2013.217. [Epub ahead of print].

Radiation sickness

Blockade of CD47 increases survival of mice exposed to lethal total body irradiation.
Soto-Pantoja DR, Ridnour LA, Wink DA, Roberts DD. Sci Reports. 2013;3:1038. doi:10.1038/srep01038.

Infectious diseases



Gene-Silencing Antisense Oligomers Inhibit Acinetobacter Growth In Vitro and In Vivo.
Geller BL, Marshall-Batty K, Schnell FJ, McKnight MM, Iversen PL, Greenberg DE. J Infect Dis. 2013;[Epub ahead of print] doi:10.1093/infdis/jit460.


Peptide Conjugated Phosphorodiamidate Morpholino Oligomers Increase Survival of Mice Challenged with Ames Bacillus anthracis.
Panchal RG, Geller BL, Mellbye B, Lane D, Iversen PL, Bavari S. Nucleic Acid Ther. 2012 Oct;22(5):316-22. doi: 10.1089/nat.2012.0362. Epub 2012 Sep 14.


Antisense Phosphorodiamidate Morpholino Oligomers Targeted to an Essential Gene Inhibit Burkholderia cepacia Complex.
Greenberg DE, Marshall-Batty KR, Brinster LR, Zarember KA, Shaw PA, Mellbye BL, Iversen PL, Holland SM, Geller BL. J Infect Dis. 2010 May 3. [Epub ahead of print]


Cationic phosphorodiamidate morpholino oligomers efficiently prevent growth of Escherichia coli in vitro and in vivo.
Mellbye BL, Weller DD, Hassinger JN, Reeves MD, Lovejoy CE, Iversen PL, Geller BL. J Antimicrob Chemother. 2009 Nov 1. [Epub ahead of print]


Inhibition of Intracellular Growth of Salmonella enterica serovar typhimurium in Tissue Culture by Antisense Peptide-Phosphorodiamidate Morpholino Oligomer.
Mitev GM, Mellbye BL, Iversen PL, Geller BL. Antimicrob Agents Chemother. 2009 Jul 6. [Epub ahead of print]


A peptide-morpholino oligomer conjugate targeting Staphylococcus aureus gyrA mRNA improves healing in an infected mouse cutaneous wound model.
Sawyer AJ, Wesolowski D, Gandotra N, Stojadinovic A, Izadjoo M, Altman S, Kyriakides TR. Int J Pharmaceutics. 2013;[Epub ahead of print] doi:10.1016/j.ijpharm.2013.05.041



Inhibition of alphavirus infection in cell culture and in mice with antisense morpholino oligomers.
Paessler S, Rijnbrand R, Stein DA, Ni H, Yun NE, Dziuba N, Borisevich V, Seregin A, Ma Y, Blouch R, Iversen PL, Zacks MA. Virology. 2008 Jul 5;376(2):357-70. Epub 2008 May 12.


Development of peptide-conjugated morpholino oligomers as pan-arenavirus inhibitors.
Neuman BW, Bederka LH, Stein DA, Ting JP, Moulton HM, Buchmeier MJ. Antimicrob Agents Chemother. 2011 Aug 8. [Epub ahead of print].


Antiviral Phosphorodiamidate Morpholino Oligomers are Protective against Chikungunya Virus Infection on Cell-based and Murine Models.
Lam S, Chen H, Chen CK, Min N, Chu JJH. Sci Rep. 2015;5:12727 doi:10.1038/srep12727.


Safety and Pharmacokinetic Profiles of Phosphorodiamidate Morpholino Oligomers with Activity against Ebola Virus and Marburg Virus: Results of Two Single Ascending Dose Studies.
Heald AE, Iversen PL, Saoud JB, Sazani P, Charleston JS, Axtelle T, Wong M, Smith WB, Vutikullird A, Kaye E. Antimicrob Agents Chemother. 2014 Aug 25. pii: AAC.03442-14. [Epub ahead of print].

AVI-7288 for Marburg Virus in Nonhuman Primates and Humans.
Heald AE, Charleston JS, Iversen PL, Warren TK, Saoud JB, Al-Ibrahim M, Wells JW, Warfield KL, Swenson DL, Welch LS, Sazani P, Wong M, Berry D, Kaye EM, Bavari S. New Engl J Med. 2015;373:339-48. doi:10.1056/NEJMoa1410345.

A single phosphorodiamidate morpholino oligomer targeting VP24 protects rhesus monkeys against lethal Ebola virus infection.
Warren TK, Whitehouse CA, Wells J, Welch L, Heald AE, Charleston JS, Sazani P, Reid SP, Iversen PL, Bavari S. mBio. 2015;6(1):e02344-14. doi:10.1128/mBio.02344-14.


Inhibition of enterovirus 71 infection by antisense octaguanidinium dendrimer-conjugated morpholino oligomers.
Tan CW, Chan YF, Quah YW, Poh CL. I Antiviral Res. 2014;[Epub ahead of print] doi:10.1016/j.antiviral.2014.04.004.


Treatment of AG129 mice with antisense morpholino oligomers increases survival time following challenge with dengue 2 virus.
Stein DA, Huang CY, Silengo S, Amantana A, Crumley S, Blouch RE, Iversen PL, Kinney RM. J Antimicrob Chemother. 2008 Sep;62(3):555-65. Epub 2008 Jun 19.

Inhibition of dengue virus serotypes 1 to 4 in vero cell cultures with morpholino oligomers.
Kinney RM, Huang CY, Rose BC, Kroeker AD, Dreher TW, Iversen PL, Stein DA. J Virol. 2005 Apr;79(8):5116-28.

Hepatitis C

Hepatitis C Virus Internal Ribosome Entry Site (IRES) Stem Loop IIId Contains a Phylogenetically Conserved GGG Triplet Essential for Translation and IRES Folding.
Jubin R, Vantuno NE, Kieft JS, Murray MG, Doudna JA, Lau JY, Baroudy BM. J Virol 2000 Nov 15;74(22):10430-10437.

Hepatitis E

Inhibition of hepatitis E virus replication by peptide-conjugated morpholino oligomers.
Nan Y, Ma Z, Kannan H, Stein DA, Iversen PI, Meng X-J, Zhang Y-J. Antiviral Res. 2015;[Epub ahead of print] doi:10.1016/j.antiviral.2015.06.006.


Inhibition of primary effusion lymphoma engraftment in SCID mice by morpholino oligomers against early lytic genes of Kaposi’s sarcoma-associated herpesvirus.
Zhang YJ, Patel D, Nan Y, Fan S. Antivir Ther. 2011;16(5):657-66.

Reduction of herpes simplex virus type-2 replication in cell cultures and in rodent models with peptide-conjugated morpholino oligomers.
Eide K, Moerdyk-Schauwecker M, Stein DA, Bildfell R, Koelle DM, Jin L. Antivir Ther. 2010;15(8):1141-1149.

Blockade of viral interleukin-6 expression of Kaposi’s sarcoma-associated herpesvirus.
Zhang YJ, Bonaparte RS, Patel D, Stein DA, Iversen PL. Mol Cancer Ther. 2008 Mar;7(3):712-20.


Inhibition of influenza virus infection in human airway cell cultures by an antisense peptide-conjugated morpholino oligomer targeting the hemagglutinin activating protease TMPRSS2.
Böttcher-Friebertshäuser E, Stein DA, Klenk HD, Garten W. J Virol. 2010 Dec 1. [Epub ahead of print].

Inhibition of influenza A H3N8 virus infections in mice by morpholino oligomers.
Lupfer C, Stein DA, Mourich DV, Tepper SE, Iversen PL, Pastey M. Arch Virol. 2008 May;153(5):929-37. Epub 2008 Mar 28.

Morpholino oligomers targeting the PB1 and NP genes enhance the survival of mice infected with highly pathogenic influenza A H7N7 virus.
Gabriel G, Nordmann A, Stein DA, Iversen PL, Klenk HD. J Gen Virol. 2008 Apr;89(Pt 4):939-48.

Japanese Encephalitis

Antiviral and Neuroprotective Role of Octaguanidinium Dendrimer-Conjugated Morpholino Oligomers in Japanese Encephalitis.
Nazmi A, Dutta K, Basu A. PLoS Negl Trop Dis 2010;4(11): e892. doi:10.1371/journal.pntd.0000892.

Inhibition of Japanese encephalitis virus replication in cultured cells and mice by a peptide-conjugated morpholino oligomer.
Anantpadma M, Stein DA, Vrati S. J Antimicrob Chemother. 2010 Mar 18. [Epub ahead of print].


Inhibition of measles virus infections in cell cultures by peptide-conjugated morpholino oligomers.
Sleeman K, Stein DA, Tamin A, Reddish M, Iversen PL, Rota PA. Virus Res. 2008 Dec 4. [Epub ahead of print].


Inhibition of norovirus replication by morpholino oligomers targeting the 5′-end of the genome.
Bok K, Cavanaugh VJ, Matson DO, González-Molleda L, Chang KO, Zintz C, Smith AW, Iversen P, Green KY, Campbell AE. Virology. 2008 Sep 8. [Epub ahead of print].

Respiratory syncytial virus

Inhibition of Respiratory Syncytial Virus Infections With Morpholino Oligomers in Cell Cultures and in Mice.
Lai SH, Stein DA, Guerrero-Plata A, Liao SL, Ivanciuc T, Hong C, Iversen PL, Casola A, Garofalo RP. Mol Ther. 2008 Apr 29; [Epub ahead of print] .


Inhibition, escape, and attenuated growth of severe acute respiratory syndrome coronavirus treated with antisense morpholino oligomers.
Neuman BW, Stein DA, Kroeker AD, Churchill MJ, Kim AM, Kuhn P, Dawson P, Moulton HM, Bestwick RK, Iversen PL, Buchmeier MJ. J Virol. 2005 Aug;79(15):9665-76.

West Nile

In vitro resistance selection and in vivo efficacy of morpholino oligomers against West Nile virus.
Deas TS, Bennett CJ, Jones SA, Tilgner M, Ren P, Behr MJ, Stein DA, Iversen PL, Kramer LD, Bernard KA, Shi PY. Antimicrob Agents Chemother. 2007 Jul;51(7):2470-82. Epub 2007 May 7.

Inhibition of flavivirus infections by antisense oligomers specifically suppressing viral translation and RNA replication.
Deas TS, Binduga-Gajewska I, Tilgner M, Ren P, Stein DA, Moulton HM, Iversen PL, Kauffman EB, Kramer LD, Shi PY. J Virol. 2005 Apr;79(8):4599-609.



Glucosylceramide transferase activity is critical for encystation and viable cyst production by an intestinal protozoan, Giardia lamblia.
Mendez TL, Chatterjee AD, Duarte TT, Gazos-Lopes F, Robles-Martinez L, Roy D, Sun J, Maldonado RA, Roychowdhury S, Almeida IC, Das S. J Biol Chem. 2013;[Epub ahead of print] doi:10.1074/jbc.M112.438416.

Using morpholinos for gene knockdown in Giardia intestinalis.
Carpenter ML, Cande WZ. Eukaryot Cell. 2009 Apr 17. [Epub ahead of print].


Gene selective mRNA cleavage inhibits the development of Plasmodium falciparum.
Augagneur Y, Wesolowski D, Tae HS, Altman S, Mamoun CB. PNAS. 2012;[Epub ahead of print] doi:10.1073/pnas.1203516109.


Silencing of GRA10 protein expression inhibits Toxoplasma gondii intracellular growth and development.
Witola WH, Bauman B, McHugh M, Matthews K. Parasitol Int. 2014 May 13. pii: S1383-5769(14)00059-2. doi: 10.1016/j.parint.2014.05.001. [Epub ahead of print]

Molecular target validation, antimicrobial delivery, and potential treatment of Toxoplasma gondii infections.
Lai BS, Witola WH, El Bissati KE, Zhou Y, Mui E, Fomovska A, McLeod R. PNAS. 2012;[Epub ahead of print] doi:10.1073/pnas.1208775109.