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A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

Yin H, Moulton HM, Betts C, Seow Y, Boutilier J, Iverson PL, Wood MJA.

Hum Mol Genet. 2019 Feb 15;28(4):699. doi: 10.1093/hmg/ddy377. No abstract available.


Anchor peptide captures, targets, and loads exosomes of diverse origins for diagnostics and therapy.

Gao X, Ran N, Dong X, Zuo B, Yang R, Zhou Q, Moulton HM, Seow Y, Yin H.

Sci Transl Med. 2018 Jun 6;10(444). pii: eaat0195. doi: 10.1126/scitranslmed.aat0195. Erratum in: Sci Transl Med. 2018 Nov 28;10(469):.


Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.

Echigoya Y, Nakamura A, Nagata T, Urasawa N, Lim KRQ, Trieu N, Panesar D, Kuraoka M, Moulton HM, Saito T, Aoki Y, Iversen P, Sazani P, Kole R, Maruyama R, Partridge T, Takeda S, Yokota T.

Proc Natl Acad Sci U S A. 2017 Apr 18;114(16):4213-4218. doi: 10.1073/pnas.1613203114. Epub 2017 Apr 3.


Surface Plasmon Resonance-Based Concentration Determination Assay: Label-Free and Antibody-Free Quantification of Morpholinos.

Boutilier J, Moulton HM.

Methods Mol Biol. 2017;1565:251-263. doi: 10.1007/978-1-4939-6817-6_21.


Aggregation and Disaggregation of Morpholino Oligomers in Solution.

Chow G, Morcos PA, Moulton HM.

Methods Mol Biol. 2017;1565:31-38. doi: 10.1007/978-1-4939-6817-6_3.


Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice.

Cao L, Han G, Lin C, Gu B, Gao X, Moulton HM, Seow Y, Yin H.

Mol Ther Nucleic Acids. 2016 Jun 28;5(6):e329. doi: 10.1038/mtna.2016.46.


Meta- and Orthogonal Integration of Influenza "OMICs" Data Defines a Role for UBR4 in Virus Budding.

Tripathi S, Pohl MO, Zhou Y, Rodriguez-Frandsen A, Wang G, Stein DA, Moulton HM, DeJesus P, Che J, Mulder LC, Yángüez E, Andenmatten D, Pache L, Manicassamy B, Albrecht RA, Gonzalez MG, Nguyen Q, Brass A, Elledge S, White M, Shapira S, Hacohen N, Karlas A, Meyer TF, Shales M, Gatorano A, Johnson JR, Jang G, Johnson T, Verschueren E, Sanders D, Krogan N, Shaw M, König R, Stertz S, García-Sastre A, Chanda SK.

Cell Host Microbe. 2015 Dec 9;18(6):723-35. doi: 10.1016/j.chom.2015.11.002.


The zebrafish, Danio rerio, as a model for Toxoplasma gondii: an initial description of infection in fish.

Sanders JL, Zhou Y, Moulton HM, Moulton ZX, McLeod R, Dubey JP, Weiss LM, Kent ML.

J Fish Dis. 2015 Jul;38(7):675-9. doi: 10.1111/jfd.12393. Epub 2015 May 7. No abstract available.


Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice.

Wu B, Cloer C, Lu P, Milazi S, Shaban M, Shah SN, Marston-Poe L, Moulton HM, Lu QL.

Gene Ther. 2014 Sep;21(9):785-93. doi: 10.1038/gt.2014.53. Epub 2014 Jun 19.


Unanchored K48-linked polyubiquitin synthesized by the E3-ubiquitin ligase TRIM6 stimulates the interferon-IKKε kinase-mediated antiviral response.

Rajsbaum R, Versteeg GA, Schmid S, Maestre AM, Belicha-Villanueva A, Martínez-Romero C, Patel JR, Morrison J, Pisanelli G, Miorin L, Laurent-Rolle M, Moulton HM, Stein DA, Fernandez-Sesma A, tenOever BR, García-Sastre A.

Immunity. 2014 Jun 19;40(6):880-95. doi: 10.1016/j.immuni.2014.04.018. Epub 2014 May 29.


Effective dystrophin restoration by a novel muscle-homing peptide-morpholino conjugate in dystrophin-deficient mdx mice.

Gao X, Zhao J, Han G, Zhang Y, Dong X, Cao L, Wang Q, Moulton HM, Yin H.

Mol Ther. 2014 Jul;22(7):1333-1341. doi: 10.1038/mt.2014.63. Epub 2014 Apr 15.


Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy Δ7 mouse model phenotype.

Nizzardo M, Simone C, Salani S, Ruepp MD, Rizzo F, Ruggieri M, Zanetta C, Brajkovic S, Moulton HM, Müehlemann O, Bresolin N, Comi GP, Corti S.

Clin Ther. 2014 Mar 1;36(3):340-56.e5. doi: 10.1016/j.clinthera.2014.02.004.


Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency.

Yin H, Boisguerin P, Moulton HM, Betts C, Seow Y, Boutilier J, Wang Q, Walsh A, Lebleu B, Wood MJ.

Mol Ther Nucleic Acids. 2013 Sep 24;2:e124. doi: 10.1038/mtna.2013.51.


In vivo delivery of morpholino oligos by cell-penetrating peptides.

Moulton HM.

Curr Pharm Des. 2013;19(16):2963-9. Review.


Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino.

Wu B, Lu P, Cloer C, Shaban M, Grewal S, Milazi S, Shah SN, Moulton HM, Lu QL.

Am J Pathol. 2012 Aug;181(2):392-400. doi: 10.1016/j.ajpath.2012.04.006. Epub 2012 Jun 7.


Cell-penetrating peptides enhance systemic delivery of antisense morpholino oligomers.

Moulton HM.

Methods Mol Biol. 2012;867:407-14. doi: 10.1007/978-1-61779-767-5_26.


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 Oct;55(10):4631-8. doi: 10.1128/AAC.00650-11. Epub 2011 Aug 8.


Diaphragm rescue alone prevents heart dysfunction in dystrophic mice.

Crisp A, Yin H, Goyenvalle A, Betts C, Moulton HM, Seow Y, Babbs A, Merritt T, Saleh AF, Gait MJ, Stuckey DJ, Clarke K, Davies KE, Wood MJ.

Hum Mol Genet. 2011 Feb 1;20(3):413-21. doi: 10.1093/hmg/ddq477. Epub 2010 Nov 9.


Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO.

Yin H, Moulton HM, Betts C, Merritt T, Seow Y, Ashraf S, Wang Q, Boutilier J, Wood MJ.

Mol Ther. 2010 Oct;18(10):1822-9. doi: 10.1038/mt.2010.151. Epub 2010 Aug 10.


Dystrophin isoform induction in vivo by antisense-mediated alternative splicing.

Fletcher S, Adams AM, Johnsen RD, Greer K, Moulton HM, Wilton SD.

Mol Ther. 2010 Jun;18(6):1218-23. doi: 10.1038/mt.2010.45. Epub 2010 Mar 23.


Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy.

Moulton HM, Moulton JD.

Biochim Biophys Acta. 2010 Dec;1798(12):2296-303. doi: 10.1016/j.bbamem.2010.02.012. Epub 2010 Feb 17.


Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.

Jearawiriyapaisarn N, Moulton HM, Sazani P, Kole R, Willis MS.

Cardiovasc Res. 2010 Feb 1;85(3):444-53. doi: 10.1093/cvr/cvp335. Epub 2009 Oct 8.


Peptide-morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophy.

Moulton HM, Wu B, Jearawiriyapaisarn N, Sazani P, Lu QL, Kole R.

Ann N Y Acad Sci. 2009 Sep;1175:55-60. doi: 10.1111/j.1749-6632.2009.04976.x.


A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

Yin H, Moulton HM, Betts C, Seow Y, Boutilier J, Iverson PL, Wood MJ.

Hum Mol Genet. 2009 Nov 15;18(22):4405-14. doi: 10.1093/hmg/ddp395. Epub 2009 Aug 18.


Chemical modifications of antisense morpholino oligomers enhance their efficacy against Ebola virus infection.

Swenson DL, Warfield KL, Warren TK, Lovejoy C, Hassinger JN, Ruthel G, Blouch RE, Moulton HM, Weller DD, Iversen PL, Bavari S.

Antimicrob Agents Chemother. 2009 May;53(5):2089-99. doi: 10.1128/AAC.00936-08. Epub 2009 Feb 17.


RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice.

Svasti S, Suwanmanee T, Fucharoen S, Moulton HM, Nelson MH, Maeda N, Smithies O, Kole R.

Proc Natl Acad Sci U S A. 2009 Jan 27;106(4):1205-10. doi: 10.1073/pnas.0812436106. Epub 2009 Jan 21.


Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer.

Wu B, Moulton HM, Iversen PL, Jiang J, Li J, Li J, Spurney CF, Sali A, Guerron AD, Nagaraju K, Doran T, Lu P, Xiao X, Lu QL.

Proc Natl Acad Sci U S A. 2008 Sep 30;105(39):14814-9. doi: 10.1073/pnas.0805676105. Epub 2008 Sep 19.


Delivery of steric block morpholino oligomers by (R-X-R)4 peptides: structure-activity studies.

Abes R, Moulton HM, Clair P, Yang ST, Abes S, Melikov K, Prevot P, Youngblood DS, Iversen PL, Chernomordik LV, Lebleu B.

Nucleic Acids Res. 2008 Nov;36(20):6343-54. doi: 10.1093/nar/gkn541. Epub 2008 Sep 16.


Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function.

Yin H, Moulton HM, Seow Y, Boyd C, Boutilier J, Iverson P, Wood MJ.

Hum Mol Genet. 2008 Dec 15;17(24):3909-18. doi: 10.1093/hmg/ddn293. Epub 2008 Sep 10.


Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.

Jearawiriyapaisarn N, Moulton HM, Buckley B, Roberts J, Sazani P, Fucharoen S, Iversen PL, Kole R.

Mol Ther. 2008 Sep;16(9):1624-9. doi: 10.1038/mt.2008.120. Epub 2008 Jun 10.


Cell penetrating peptide conjugates of steric block oligonucleotides.

Lebleu B, Moulton HM, Abes R, Ivanova GD, Abes S, Stein DA, Iversen PL, Arzumanov AA, Gait MJ.

Adv Drug Deliv Rev. 2008 Mar 1;60(4-5):517-29. Epub 2007 Oct 22. Review.


Arginine-rich cell-penetrating peptides facilitate delivery of antisense oligomers into murine leukocytes and alter pre-mRNA splicing.

Marshall NB, Oda SK, London CA, Moulton HM, Iversen PL, Kerkvliet NI, Mourich DV.

J Immunol Methods. 2007 Aug 31;325(1-2):114-26. Epub 2007 Jul 27.


Cell-penetrating peptides as transporters for morpholino oligomers: effects of amino acid composition on intracellular delivery and cytotoxicity.

Wu RP, Youngblood DS, Hassinger JN, Lovejoy CE, Nelson MH, Iversen PL, Moulton HM.

Nucleic Acids Res. 2007;35(15):5182-91. Epub 2007 Aug 1.


Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.

Moulton HM, Fletcher S, Neuman BW, McClorey G, Stein DA, Abes S, Wilton SD, Buchmeier MJ, Lebleu B, Iversen PL.

Biochem Soc Trans. 2007 Aug;35(Pt 4):826-8. Review.


Cell-penetrating-peptide-based delivery of oligonucleotides: an overview.

Abes R, Arzumanov AA, Moulton HM, Abes S, Ivanova GD, Iversen PL, Gait MJ, Lebleu B.

Biochem Soc Trans. 2007 Aug;35(Pt 4):775-9. Review.


Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate.

Amantana A, Moulton HM, Cate ML, Reddy MT, Whitehead T, Hassinger JN, Youngblood DS, Iversen PL.

Bioconjug Chem. 2007 Jul-Aug;18(4):1325-31. Epub 2007 Jun 21.


Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse.

Fletcher S, Honeyman K, Fall AM, Harding PL, Johnsen RD, Steinhaus JP, Moulton HM, Iversen PL, Wilton SD.

Mol Ther. 2007 Sep;15(9):1587-92. Epub 2007 Jun 19.


Chemical modifications to improve the cellular uptake of oligonucleotides.

Debart F, Abes S, Deglane G, Moulton HM, Clair P, Gait MJ, Vasseur JJ, Lebleu B.

Curr Top Med Chem. 2007;7(7):727-37. Review.


Antiviral effects of antisense morpholino oligomers in murine coronavirus infection models.

Burrer R, Neuman BW, Ting JP, Stein DA, Moulton HM, Iversen PL, Kuhn P, Buchmeier MJ.

J Virol. 2007 Jun;81(11):5637-48. Epub 2007 Mar 7.


Stability of cell-penetrating peptide-morpholino oligomer conjugates in human serum and in cells.

Youngblood DS, Hatlevig SA, Hassinger JN, Iversen PL, Moulton HM.

Bioconjug Chem. 2007 Jan-Feb;18(1):50-60.


Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents.

Abes S, Moulton HM, Clair P, Prevot P, Youngblood DS, Wu RP, Iversen PL, Lebleu B.

J Control Release. 2006 Dec 1;116(3):304-13. Epub 2006 Sep 30.


Inhibition and escape of SARS-CoV treated with antisense morpholino oligomers.

Neuman BW, Stein DA, Kroeker AD, Moulton HM, Bestwick RK, Iversen PL, Buchmeier MJ.

Adv Exp Med Biol. 2006;581:567-71. Review. No abstract available.


Inhibition of coxsackievirus B3 in cell cultures and in mice by peptide-conjugated morpholino oligomers targeting the internal ribosome entry site.

Yuan J, Stein DA, Lim T, Qiu D, Coughlin S, Liu Z, Wang Y, Blouch R, Moulton HM, Iversen PL, Yang D.

J Virol. 2006 Dec;80(23):11510-9. Epub 2006 Sep 20.


Induced dystrophin exon skipping in human muscle explants.

McClorey G, Fall AM, Moulton HM, Iversen PL, Rasko JE, Ryan M, Fletcher S, Wilton SD.

Neuromuscul Disord. 2006 Oct;16(9-10):583-90. Epub 2006 Aug 21.


Inhibition of replication and transcription activator and latency-associated nuclear antigen of Kaposi's sarcoma-associated herpesvirus by morpholino oligomers.

Zhang YJ, Wang KY, Stein DA, Patel D, Watkins R, Moulton HM, Iversen PL, Matson DO.

Antiviral Res. 2007 Jan;73(1):12-23. Epub 2006 Jun 14.


Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD.

McClorey G, Moulton HM, Iversen PL, Fletcher S, Wilton SD.

Gene Ther. 2006 Oct;13(19):1373-81. Epub 2006 May 25.


Inhibition of infectious haematopoietic necrosis virus in cell cultures with peptide-conjugated morpholino oligomers.

Alonso M, Stein DA, Thomann E, Moulton HM, Leong JC, Iversen P, Mourich DV.

J Fish Dis. 2005 Jul;28(7):399-410.


Arginine-rich peptide conjugation to morpholino oligomers: effects on antisense activity and specificity.

Nelson MH, Stein DA, Kroeker AD, Hatlevig SA, Iversen PL, Moulton HM.

Bioconjug Chem. 2005 Jul-Aug;16(4):959-66.


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.


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.

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