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Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment.

Gait MJ, Arzumanov AA, McClorey G, Godfrey C, Betts C, Hammond S, Wood MJA.

Nucleic Acid Ther. 2019 Feb;29(1):1-12. doi: 10.1089/nat.2018.0747. Epub 2018 Oct 16.


Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment.

Betts CA, McClorey G, Healicon R, Hammond SM, Manzano R, Muses S, Ball V, Godfrey C, Merritt TM, van Westering T, O'Donovan L, Wells KE, Gait MJ, Wells DJ, Tyler D, Wood MJ.

Hum Mol Genet. 2019 Feb 1;28(3):396-406. doi: 10.1093/hmg/ddy346.


Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy.

Blain AM, Greally E, McClorey G, Manzano R, Betts CA, Godfrey C, O'Donovan L, Coursindel T, Gait MJ, Wood MJ, MacGowan GA, Straub VW.

PLoS One. 2018 Jun 18;13(6):e0198897. doi: 10.1371/journal.pone.0198897. eCollection 2018.


Solid-Phase Synthesis of Difficult Purine-Rich PNAs through Selective Hmb Incorporation: Application to the Total Synthesis of Cell Penetrating Peptide-PNAs.

Tailhades J, Takizawa H, Gait MJ, Wellings DA, Wade JD, Aoki Y, Shabanpoor F.

Front Chem. 2017 Oct 17;5:81. doi: 10.3389/fchem.2017.00081. eCollection 2017.


ClickIn: a flexible protocol for quantifying mitochondrial uptake of nucleobase derivatives.

Hoogewijs K, James AM, Smith RA, Abendroth F, Gait MJ, Murphy MP, Lightowlers RN.

Interface Focus. 2017 Apr 6;7(2):20160117. doi: 10.1098/rsfs.2016.0117.


Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy.

Shabanpoor F, Hammond SM, Abendroth F, Hazell G, Wood MJA, Gait MJ.

Nucleic Acid Ther. 2017 Jun;27(3):130-143. doi: 10.1089/nat.2016.0652. Epub 2017 Jan 24.


Corrigendum: Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics.

Coenen-Stass AM, McClorey G, Manzano R, Betts CA, Blain A, Saleh AF, Gait MJ, Lochmüller H, Wood MJ, Roberts TC.

Sci Rep. 2016 Dec 19;6:37047. doi: 10.1038/srep37047. No abstract available.


Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.

Hammond SM, Hazell G, Shabanpoor F, Saleh AF, Bowerman M, Sleigh JN, Meijboom KE, Zhou H, Muntoni F, Talbot K, Gait MJ, Wood MJ.

Proc Natl Acad Sci U S A. 2016 Sep 27;113(39):10962-7. doi: 10.1073/pnas.1605731113. Epub 2016 Sep 12.


Assessing the Delivery of Molecules to the Mitochondrial Matrix Using Click Chemistry.

Hoogewijs K, James AM, Smith RA, Gait MJ, Murphy MP, Lightowlers RN.

Chembiochem. 2016 Jul 15;17(14):1312-6. doi: 10.1002/cbic.201600188. Epub 2016 May 25.


Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics.

Coenen-Stass AM, McClorey G, Manzano R, Betts CA, Blain A, Saleh AF, Gait MJ, Lochmüller H, Wood MJ, Roberts TC.

Sci Rep. 2015 Nov 23;5:17014. doi: 10.1038/srep17014. Erratum in: Sci Rep. 2016 Dec 19;6:37047.


Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration.

Roberts TC, Johansson HJ, McClorey G, Godfrey C, Blomberg KE, Coursindel T, Gait MJ, Smith CI, Lehtiö J, El Andaloussi S, Wood MJ.

Hum Mol Genet. 2015 Dec 1;24(23):6756-68. doi: 10.1093/hmg/ddv381. Epub 2015 Sep 18.


In Vitro Assays to Assess Exon Skipping in Duchenne Muscular Dystrophy.

Boisguerin P, O'Donovan L, Gait MJ, Lebleu B.

Methods Mol Biol. 2015;1324:317-29. doi: 10.1007/978-1-4939-2806-4_20.


Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides.

Burki U, Keane J, Blain A, O'Donovan L, Gait MJ, Laval SH, Straub V.

Nucleic Acid Ther. 2015 Oct;25(5):275-84. doi: 10.1089/nat.2014.0528. Epub 2015 Jul 15.


Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy.

Betts CA, Saleh AF, Carr CA, Muses S, Wells KE, Hammond SM, Godfrey C, McClorey G, Woffindale C, Clarke K, Wells DJ, Gait MJ, Wood MJ.

Sci Rep. 2015 Jun 26;5:11632. doi: 10.1038/srep11632.


Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides.

Ezzat K, Aoki Y, Koo T, McClorey G, Benner L, Coenen-Stass A, O'Donovan L, Lehto T, Garcia-Guerra A, Nordin J, Saleh AF, Behlke M, Morris J, Goyenvalle A, Dugovic B, Leumann C, Gordon S, Gait MJ, El-Andaloussi S, Wood MJ.

Nano Lett. 2015 Jul 8;15(7):4364-73. doi: 10.1021/acs.nanolett.5b00490. Epub 2015 Jun 15.


Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.

Lu-Nguyen NB, Jarmin SA, Saleh AF, Popplewell L, Gait MJ, Dickson G.

Mol Ther. 2015 Aug;23(8):1341-1348. doi: 10.1038/mt.2015.88. Epub 2015 May 11.


How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse.

Godfrey C, Muses S, McClorey G, Wells KE, Coursindel T, Terry RL, Betts C, Hammond S, O'Donovan L, Hildyard J, El Andaloussi S, Gait MJ, Wood MJ, Wells DJ.

Hum Mol Genet. 2015 Aug 1;24(15):4225-37. doi: 10.1093/hmg/ddv155. Epub 2015 May 1.


Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.

Betts CA, Saleh AF, Carr CA, Hammond SM, Coenen-Stass AM, Godfrey C, McClorey G, Varela MA, Roberts TC, Clarke K, Gait MJ, Wood MJ.

Sci Rep. 2015 Mar 11;5:8986. doi: 10.1038/srep08986.


Delivery of therapeutic oligonucleotides with cell penetrating peptides.

Boisguérin P, Deshayes S, Gait MJ, O'Donovan L, Godfrey C, Betts CA, Wood MJ, Lebleu B.

Adv Drug Deliv Rev. 2015 Jun 29;87:52-67. doi: 10.1016/j.addr.2015.02.008. Epub 2015 Mar 4. Review.


Peptide nanoparticle delivery of charge-neutral splice-switching morpholino oligonucleotides.

Järver P, Zaghloul EM, Arzumanov AA, Saleh AF, McClorey G, Hammond SM, Hällbrink M, Langel Ü, Smith CI, Wood MJ, Gait MJ, El Andaloussi S.

Nucleic Acid Ther. 2015 Apr;25(2):65-77. doi: 10.1089/nat.2014.0511. Epub 2015 Jan 16.


Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy.

Shabanpoor F, McClorey G, Saleh AF, Järver P, Wood MJ, Gait MJ.

Nucleic Acids Res. 2015 Jan;43(1):29-39. doi: 10.1093/nar/gku1256. Epub 2014 Dec 2.


Parallel synthesis of cell-penetrating peptide conjugates of PMO toward exon skipping enhancement in Duchenne muscular dystrophy.

O'Donovan L, Okamoto I, Arzumanov AA, Williams DL, Deuss P, Gait MJ.

Nucleic Acid Ther. 2015 Feb;25(1):1-10. doi: 10.1089/nat.2014.0512. Epub 2014 Nov 20.


Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model.

Bestas B, Moreno PM, Blomberg KE, Mohammad DK, Saleh AF, Sutlu T, Nordin JZ, Guterstam P, Gustafsson MO, Kharazi S, Piątosa B, Roberts TC, Behlke MA, Wood MJ, Gait MJ, Lundin KE, El Andaloussi S, Månsson R, Berglöf A, Wengel J, Smith CI.

J Clin Invest. 2014 Sep;124(9):4067-81. doi: 10.1172/JCI76175. Epub 2014 Aug 8.


Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells.

Lehto T, Castillo Alvarez A, Gauck S, Gait MJ, Coursindel T, Wood MJ, Lebleu B, Boisguerin P.

Nucleic Acids Res. 2014 Mar;42(5):3207-17. doi: 10.1093/nar/gkt1220. Epub 2013 Dec 23.


Enhanced splice correction by 3', 5'-serinol and 2'-(ω-O-methylserinol) guarded OMe-RNA/DNA mixmers in cells.

Kotikam V, Arzumanov AA, Gait MJ, Kumar VA.

Artif DNA PNA XNA. 2013 Jul-Dec;4(3):77-83. doi: 10.4161/adna.27279. Epub 2013 Dec 2.


A chemical view of oligonucleotides for exon skipping and related drug applications.

Järver P, O'Donovan L, Gait MJ.

Nucleic Acid Ther. 2014 Feb;24(1):37-47. doi: 10.1089/nat.2013.0454. Epub 2013 Oct 30. Review. No abstract available.


Parallel synthesis and splicing redirection activity of cell-penetrating peptide conjugate libraries of a PNA cargo.

Deuss PJ, Arzumanov AA, Williams DL, Gait MJ.

Org Biomol Chem. 2013 Nov 21;11(43):7621-30. doi: 10.1039/c3ob41659c.


Development of a general methodology for labelling peptide-morpholino oligonucleotide conjugates using alkyne-azide click chemistry.

Shabanpoor F, Gait MJ.

Chem Commun (Camb). 2013 Nov 11;49(87):10260-2. doi: 10.1039/c3cc46067c.


Nucleic acids: new life, new materials.

Gait MJ, Komiyama M, Seeman NC, Seitz O, Micklefield J, Liu DR.

Org Biomol Chem. 2013 Apr 7;11(13):2058-9. doi: 10.1039/c3ob90017g. No abstract available.


Expression analysis in multiple muscle groups and serum reveals complexity in the microRNA transcriptome of the mdx mouse with implications for therapy.

Roberts TC, Blomberg KE, McClorey G, El Andaloussi S, Godfrey C, Betts C, Coursindel T, Gait MJ, Smith CI, Wood MJ.

Mol Ther Nucleic Acids. 2012 Aug 14;1:e39. doi: 10.1038/mtna.2012.26.


Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.

Betts C, Saleh AF, Arzumanov AA, Hammond SM, Godfrey C, Coursindel T, Gait MJ, Wood MJ.

Mol Ther Nucleic Acids. 2012 Aug 14;1:e38. doi: 10.1038/mtna.2012.30.


Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA.

Järver P, Coursindel T, Andaloussi SE, Godfrey C, Wood MJ, Gait MJ.

Mol Ther Nucleic Acids. 2012 Jun 12;1:e27. doi: 10.1038/mtna.2012.18. No abstract available.


Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.

Malerba A, Kang JK, McClorey G, Saleh AF, Popplewell L, Gait MJ, Wood MJ, Dickson G.

Mol Ther Nucleic Acids. 2012 Dec 18;1:e62. doi: 10.1038/mtna.2012.54.


Overview of alternative oligonucleotide chemistries for exon skipping.

Saleh AF, Arzumanov AA, Gait MJ.

Methods Mol Biol. 2012;867:365-78. doi: 10.1007/978-1-61779-767-5_23. Review.


Peptide-based in vivo delivery agents for oligonucleotides and siRNA.

Coursindel T, Järver P, Gait MJ.

Nucleic Acid Ther. 2012 Apr;22(2):71-6. doi: 10.1089/nat.2012.0341. Epub 2012 Mar 12. No abstract available.


Exploiting cell surface thiols to enhance cellular uptake.

Torres AG, Gait MJ.

Trends Biotechnol. 2012 Apr;30(4):185-90. doi: 10.1016/j.tibtech.2011.12.002. Epub 2012 Jan 17.


Synthesis and biological activity of phosphonoacetate- and thiophosphonoacetate-modified 2'-O-methyl oligoribonucleotides.

Threlfall RN, Torres AG, Krivenko A, Gait MJ, Caruthers MH.

Org Biomol Chem. 2012 Jan 28;10(4):746-54. doi: 10.1039/c1ob06614e. Epub 2011 Nov 29.


Chemical structure requirements and cellular targeting of microRNA-122 by peptide nucleic acids anti-miRs.

Torres AG, Fabani MM, Vigorito E, Williams D, Al-Obaidi N, Wojciechowski F, Hudson RH, Seitz O, Gait MJ.

Nucleic Acids Res. 2012 Mar;40(5):2152-67. doi: 10.1093/nar/gkr885. Epub 2011 Nov 8.


Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.

Yin H, Saleh AF, Betts C, Camelliti P, Seow Y, Ashraf S, Arzumanov A, Hammond S, Merritt T, Gait MJ, Wood MJ.

Mol Ther. 2011 Jul;19(7):1295-303. doi: 10.1038/mt.2011.79. Epub 2011 Apr 19.


MicroRNA fate upon targeting with anti-miRNA oligonucleotides as revealed by an improved Northern-blot-based method for miRNA detection.

Torres AG, Fabani MM, Vigorito E, Gait MJ.

RNA. 2011 May;17(5):933-43. doi: 10.1261/rna.2533811. Epub 2011 Mar 25.


PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation.

Ezzat K, Andaloussi SE, Zaghloul EM, Lehto T, Lindberg S, Moreno PM, Viola JR, Magdy T, Abdo R, Guterstam P, Sillard R, Hammond SM, Wood MJ, Arzumanov AA, Gait MJ, Smith CI, Hällbrink M, Langel Ü.

Nucleic Acids Res. 2011 Jul;39(12):5284-98. doi: 10.1093/nar/gkr072. Epub 2011 Feb 23.


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.


Splice redirection as a convenient assay to monitor CPP-ON efficiency and mechanism.

Abes R, Arzumanov AA, Saleh AF, Said Hassane F, Gait MJ, Lebleu B.

Methods Mol Biol. 2011;683:307-20. doi: 10.1007/978-1-60761-919-2_22.


Synthesis and splice-redirecting activity of branched, arginine-rich peptide dendrimer conjugates of peptide nucleic acid oligonucleotides.

Saleh AF, Arzumanov A, Abes R, Owen D, Lebleu B, Gait MJ.

Bioconjug Chem. 2010 Oct 20;21(10):1902-11. doi: 10.1021/bc100275r.


In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.

Wang Q, Yin H, Camelliti P, Betts C, Moulton H, Lee H, Saleh AF, Gait MJ, Wood MJ.

J Gene Med. 2010 Apr;12(4):354-64. doi: 10.1002/jgm.1446.


Efficient inhibition of miR-155 function in vivo by peptide nucleic acids.

Fabani MM, Abreu-Goodger C, Williams D, Lyons PA, Torres AG, Smith KG, Enright AJ, Gait MJ, Vigorito E.

Nucleic Acids Res. 2010 Jul;38(13):4466-75. doi: 10.1093/nar/gkq160. Epub 2010 Mar 11.


RNA-targeted splice-correction therapy for neuromuscular disease.

Wood MJ, Gait MJ, Yin H.

Brain. 2010 Apr;133(Pt 4):957-72. doi: 10.1093/brain/awq002. Epub 2010 Feb 11. Review.


Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse.

Yin H, Betts C, Saleh AF, Ivanova GD, Lee H, Seow Y, Kim D, Gait MJ, Wood MJ.

Mol Ther. 2010 Apr;18(4):819-27. doi: 10.1038/mt.2009.310. Epub 2010 Jan 12.


Cell penetrating peptides: overview and applications to the delivery of oligonucleotides.

Said Hassane F, Saleh AF, Abes R, Gait MJ, Lebleu B.

Cell Mol Life Sci. 2010 Mar;67(5):715-26. doi: 10.1007/s00018-009-0186-0. Epub 2009 Nov 7. Review.


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