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Isolation, structural identification, synthesis, and pharmacological profiling of 1,2-trans-dihydro-1,2-diol metabolites of the utrophin modulator ezutromid.

Chatzopoulou M, Claridge TDW, Davies KE, Davies SG, Elsey D, Emer E, Fletcher AM, Harriman S, Robinson N, Rowley J, Russell AJ, Tinsley J, Weaver R, Wilkinson I, Willis N, Wilson F, Wynne GM.

J Med Chem. 2019 Oct 10. doi: 10.1021/acs.jmedchem.9b01547. [Epub ahead of print]


Surrogate gene therapy for muscular dystrophy.

Davies KE, Chamberlain JS.

Nat Med. 2019 Oct 7. doi: 10.1038/s41591-019-0604-2. [Epub ahead of print] No abstract available.


Limitations to adaptive homeostasis in an hyperoxia-induced model of accelerated ageing.

Pomatto LCD, Sun PY, Yu K, Gullapalli S, Bwiza CP, Sisliyan C, Wong S, Zhang H, Forman HJ, Oliver PL, Davies KE, Davies KJA.

Redox Biol. 2019 Jun;24:101194. doi: 10.1016/j.redox.2019.101194. Epub 2019 Apr 14.


The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy.

Guiraud S, Edwards B, Babbs A, Squire SE, Berg A, Moir L, Wood MJ, Davies KE.

Hum Mol Genet. 2019 Jul 1;28(13):2189-2200. doi: 10.1093/hmg/ddz049.


Regenerative biomarkers for Duchenne muscular dystrophy.

Guiraud S, Davies KE.

Neural Regen Res. 2019 Aug;14(8):1317-1320. doi: 10.4103/1673-5374.253534. Review.


SLTs' conceptions about their own and parents' roles during intervention with preschool children.

Davies KE, Marshall J, Brown LJE, Goldbart J.

Int J Lang Commun Disord. 2019 Jul;54(4):596-605. doi: 10.1111/1460-6984.12462. Epub 2019 Feb 19.


Micro-dystrophin Genes Bring Hope of an Effective Therapy for Duchenne Muscular Dystrophy.

Davies KE, Guiraud S.

Mol Ther. 2019 Mar 6;27(3):486-488. doi: 10.1016/j.ymthe.2019.01.019. Epub 2019 Feb 12. No abstract available.


A Phase 1b Trial to Assess the Pharmacokinetics of Ezutromid in Pediatric Duchenne Muscular Dystrophy Patients on a Balanced Diet.

Muntoni F, Tejura B, Spinty S, Roper H, Hughes I, Layton G, Davies KE, Harriman S, Tinsley J.

Clin Pharmacol Drug Dev. 2019 Oct;8(7):922-933. doi: 10.1002/cpdd.642. Epub 2019 Jan 16.


Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice.

Kennedy TL, Guiraud S, Edwards B, Squire S, Moir L, Babbs A, Odom G, Golebiowski D, Schneider J, Chamberlain JS, Davies KE.

Mol Ther Methods Clin Dev. 2018 Oct 16;11:92-105. doi: 10.1016/j.omtm.2018.10.005. eCollection 2018 Dec 14.


Embryonic myosin is a regeneration marker to monitor utrophin-based therapies for DMD.

Guiraud S, Edwards B, Squire SE, Moir L, Berg A, Babbs A, Ramadan N, Wood MJ, Davies KE.

Hum Mol Genet. 2019 Jan 15;28(2):307-319. doi: 10.1093/hmg/ddy353.


Absent sleep EEG spindle activity in GluA1 (Gria1) knockout mice: relevance to neuropsychiatric disorders.

Ang G, McKillop LE, Purple R, Blanco-Duque C, Peirson SN, Foster RG, Harrison PJ, Sprengel R, Davies KE, Oliver PL, Bannerman DM, Vyazovskiy VV.

Transl Psychiatry. 2018 Aug 14;8(1):154. doi: 10.1038/s41398-018-0199-2.


Alternative utrophin mRNAs contribute to phenotypic differences between dystrophin-deficient mice and Duchenne muscular dystrophy.

Perkins KJ, Davies KE.

FEBS Lett. 2018 Jun;592(11):1856-1869. doi: 10.1002/1873-3468.13099. Epub 2018 May 30.


Longitudinal investigation of neuroinflammation and metabolite profiles in the APPswe ×PS1Δe9 transgenic mouse model of Alzheimer's disease.

Chaney A, Bauer M, Bochicchio D, Smigova A, Kassiou M, Davies KE, Williams SR, Boutin H.

J Neurochem. 2018 Feb;144(3):318-335. doi: 10.1111/jnc.14251. Epub 2017 Dec 15.


Digital PCR analysis of circulating tumor DNA: a biomarker for chondrosarcoma diagnosis, prognostication, and residual disease detection.

Gutteridge A, Rathbone VM, Gibbons R, Bi M, Archard N, Davies KEJ, Brown J, Plagnol V, Pillay N, Amary F, O'Donnell P, Gupta M, Tirabosco R, Flanagan AM, Forshew T.

Cancer Med. 2017 Oct;6(10):2194-2202. doi: 10.1002/cam4.1146. Epub 2017 Aug 23.


Pharmacological advances for treatment in Duchenne muscular dystrophy.

Guiraud S, Davies KE.

Curr Opin Pharmacol. 2017 Jun;34:36-48. doi: 10.1016/j.coph.2017.04.002. Epub 2017 May 6. Review.


Identification of serum protein biomarkers for utrophin based DMD therapy.

Guiraud S, Edwards B, Squire SE, Babbs A, Shah N, Berg A, Chen H, Davies KE.

Sci Rep. 2017 Mar 2;7:43697. doi: 10.1038/srep43697.


The antioxidant protein Oxr1 influences aspects of mitochondrial morphology.

Wu Y, Davies KE, Oliver PL.

Free Radic Biol Med. 2016 Jun;95:255-67. doi: 10.1016/j.freeradbiomed.2016.03.029. Epub 2016 Mar 29.


Correlation of Utrophin Levels with the Dystrophin Protein Complex and Muscle Fibre Regeneration in Duchenne and Becker Muscular Dystrophy Muscle Biopsies.

Janghra N, Morgan JE, Sewry CA, Wilson FX, Davies KE, Muntoni F, Tinsley J.

PLoS One. 2016 Mar 14;11(3):e0150818. doi: 10.1371/journal.pone.0150818. eCollection 2016.


2015 William Allan Award.

Davies KE.

Am J Hum Genet. 2016 Mar 3;98(3):419-426. doi: 10.1016/j.ajhg.2016.01.007. No abstract available.


The Evolutionarily Conserved Tre2/Bub2/Cdc16 (TBC), Lysin Motif (LysM), Domain Catalytic (TLDc) Domain Is Neuroprotective against Oxidative Stress.

Finelli MJ, Sanchez-Pulido L, Liu KX, Davies KE, Oliver PL.

J Biol Chem. 2016 Feb 5;291(6):2751-63. doi: 10.1074/jbc.M115.685222. Epub 2015 Dec 14.


Temporal transcriptomics suggest that twin-peaking genes reset the clock.

Pembroke WG, Babbs A, Davies KE, Ponting CP, Oliver PL.

Elife. 2015 Nov 2;4. pii: e10518. doi: 10.7554/eLife.10518.


Circadian profiling in two mouse models of lysosomal storage disorders; Niemann Pick type-C and Sandhoff disease.

Richardson K, Livieratos A, Dumbill R, Hughes S, Ang G, Smith DA, Morris L, Brown LA, Peirson SN, Platt FM, Davies KE, Oliver PL.

Behav Brain Res. 2016 Jan 15;297:213-23. doi: 10.1016/j.bbr.2015.10.021. Epub 2015 Oct 20.


Advances in genetic therapeutic strategies for Duchenne muscular dystrophy.

Guiraud S, Chen H, Burns DT, Davies KE.

Exp Physiol. 2015 Dec;100(12):1458-67. doi: 10.1113/EP085308. Epub 2015 Aug 4. Review.


The Pathogenesis and Therapy of Muscular Dystrophies.

Guiraud S, Aartsma-Rus A, Vieira NM, Davies KE, van Ommen GJ, Kunkel LM.

Annu Rev Genomics Hum Genet. 2015;16:281-308. doi: 10.1146/annurev-genom-090314-025003. Epub 2015 Jun 4. Review.


Second-generation compound for the modulation of utrophin in the therapy of DMD.

Guiraud S, Squire SE, Edwards B, Chen H, Burns DT, Shah N, Babbs A, Davies SG, Wynne GM, Russell AJ, Elsey D, Wilson FX, Tinsley JM, Davies KE.

Hum Mol Genet. 2015 Aug 1;24(15):4212-24. doi: 10.1093/hmg/ddv154. Epub 2015 May 1.


The mutant Moonwalker TRPC3 channel links calcium signaling to lipid metabolism in the developing cerebellum.

Dulneva A, Lee S, Oliver PL, Di Gleria K, Kessler BM, Davies KE, Becker EB.

Hum Mol Genet. 2015 Jul 15;24(14):4114-25. doi: 10.1093/hmg/ddv150. Epub 2015 Apr 23.


Oxr1 improves pathogenic cellular features of ALS-associated FUS and TDP-43 mutations.

Finelli MJ, Liu KX, Wu Y, Oliver PL, Davies KE.

Hum Mol Genet. 2015 Jun 15;24(12):3529-44. doi: 10.1093/hmg/ddv104. Epub 2015 Mar 19.


Neuron-specific antioxidant OXR1 extends survival of a mouse model of amyotrophic lateral sclerosis.

Liu KX, Edwards B, Lee S, Finelli MJ, Davies B, Davies KE, Oliver PL.

Brain. 2015 May;138(Pt 5):1167-81. doi: 10.1093/brain/awv039. Epub 2015 Mar 9.


Preconditioning of Cardiosphere-Derived Cells With Hypoxia or Prolyl-4-Hydroxylase Inhibitors Increases Stemness and Decreases Reliance on Oxidative Metabolism.

Tan SC, Gomes RS, Yeoh KK, Perbellini F, Malandraki-Miller S, Ambrose L, Heather LC, Faggian G, Schofield CJ, Davies KE, Clarke K, Carr CA.

Cell Transplant. 2016;25(1):35-53. doi: 10.3727/096368915X687697. Epub 2015 Mar 6.


Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.

Goyenvalle A, Griffith G, Babbs A, El Andaloussi S, Ezzat K, Avril A, Dugovic B, Chaussenot R, Ferry A, Voit T, Amthor H, Bühr C, Schürch S, Wood MJ, Davies KE, Vaillend C, Leumann C, Garcia L.

Nat Med. 2015 Mar;21(3):270-5. doi: 10.1038/nm.3765. Epub 2015 Feb 2.


Laf4/Aff3, a gene involved in intellectual disability, is required for cellular migration in the mouse cerebral cortex.

Moore JM, Oliver PL, Finelli MJ, Lee S, Lickiss T, Molnár Z, Davies KE.

PLoS One. 2014 Aug 27;9(8):e105933. doi: 10.1371/journal.pone.0105933. eCollection 2014.


A novel mouse model of a patient mucolipidosis II mutation recapitulates disease pathology.

Paton L, Bitoun E, Kenyon J, Priestman DA, Oliver PL, Edwards B, Platt FM, Davies KE.

J Biol Chem. 2014 Sep 26;289(39):26709-21. doi: 10.1074/jbc.M114.586156. Epub 2014 Aug 8.


Overexpression of survival motor neuron improves neuromuscular function and motor neuron survival in mutant SOD1 mice.

Turner BJ, Alfazema N, Sheean RK, Sleigh JN, Davies KE, Horne MK, Talbot K.

Neurobiol Aging. 2014 Apr;35(4):906-15. doi: 10.1016/j.neurobiolaging.2013.09.030. Epub 2013 Oct 24.


Region-specific deficits in dopamine, but not norepinephrine, signaling in a novel A30P α-synuclein BAC transgenic mouse.

Taylor TN, Potgieter D, Anwar S, Senior SL, Janezic S, Threlfell S, Ryan B, Parkkinen L, Deltheil T, Cioroch M, Livieratos A, Oliver PL, Jennings KA, Davies KE, Ansorge O, Bannerman DM, Cragg SJ, Wade-Martins R.

Neurobiol Dis. 2014 Feb;62:193-207. doi: 10.1016/j.nbd.2013.10.005. Epub 2013 Oct 10.


Chondrolectin affects cell survival and neuronal outgrowth in in vitro and in vivo models of spinal muscular atrophy.

Sleigh JN, Barreiro-Iglesias A, Oliver PL, Biba A, Becker T, Davies KE, Becker CG, Talbot K.

Hum Mol Genet. 2014 Feb 15;23(4):855-69. doi: 10.1093/hmg/ddt477. Epub 2013 Sep 25.


Syncoilin is an intermediate filament protein in activated hepatic stellate cells.

Van Rossen E, Liu Z, Blijweert D, Eysackers N, Mannaerts I, Schroyen B, El Taghdouini A, Edwards B, Davies KE, Sokal E, Najimi M, Reynaert H, van Grunsven LA.

Histochem Cell Biol. 2014 Jan;141(1):85-99. doi: 10.1007/s00418-013-1142-5. Epub 2013 Sep 17.


AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.

Le Hir M, Goyenvalle A, Peccate C, Précigout G, Davies KE, Voit T, Garcia L, Lorain S.

Mol Ther. 2013 Aug;21(8):1551-8. doi: 10.1038/mt.2013.121. Epub 2013 Jun 11.


Cardiac α-actin over-expression therapy in dominant ACTA1 disease.

Ravenscroft G, McNamara E, Griffiths LM, Papadimitriou JM, Hardeman EC, Bakker AJ, Davies KE, Laing NG, Nowak KJ.

Hum Mol Genet. 2013 Oct 1;22(19):3987-97. doi: 10.1093/hmg/ddt252. Epub 2013 Jun 4.


Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches.

Fairclough RJ, Wood MJ, Davies KE.

Nat Rev Genet. 2013 Jun;14(6):373-8. doi: 10.1038/nrg3460. Epub 2013 Apr 23. Review.


Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.

Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G.

Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. No abstract available.


The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery.

Eckenfelder A, Tordo J, Babbs A, Davies KE, Goyenvalle A, Danos O.

Mol Ther Nucleic Acids. 2012 Jun 26;1:e31. doi: 10.1038/mtna.2012.24.


Pressure alopecia.

Davies KE, Yesudian P.

Int J Trichology. 2012 Apr;4(2):64-8. doi: 10.4103/0974-7753.96901.


Recent advances in Duchenne muscular dystrophy.

Perkins KJ, Davies KE.

Degener Neurol Neuromuscul Dis. 2012 Oct 11;2:141-164. doi: 10.2147/DNND.S26637. eCollection 2012. Review.


Spinal muscular atrophy at the crossroads of basic science and therapy.

Sleigh JN, Grice SJ, Davies KE, Talbot K.

Neuromuscul Disord. 2013 Jan;23(1):96. doi: 10.1016/j.nmd.2012.08.008. Epub 2012 Sep 13. No abstract available.


New insights into behaviour using mouse ENU mutagenesis.

Oliver PL, Davies KE.

Hum Mol Genet. 2012 Oct 15;21(R1):R72-81. Epub 2012 Aug 13. Review.


Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy.

Fairclough RJ, Perkins KJ, Davies KE.

Curr Gene Ther. 2012 Jun;12(3):206-44. Review.


Evaluating the links between schizophrenia and sleep and circadian rhythm disruption.

Pritchett D, Wulff K, Oliver PL, Bannerman DM, Davies KE, Harrison PJ, Peirson SN, Foster RG.

J Neural Transm (Vienna). 2012 Oct;119(10):1061-75. Epub 2012 May 10. Review.


Hsp72 preserves muscle function and slows progression of severe muscular dystrophy.

Gehrig SM, van der Poel C, Sayer TA, Schertzer JD, Henstridge DC, Church JE, Lamon S, Russell AP, Davies KE, Febbraio MA, Lynch GS.

Nature. 2012 Apr 4;484(7394):394-8. doi: 10.1038/nature10980.


Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.

Goyenvalle A, Babbs A, Wright J, Wilkins V, Powell D, Garcia L, Davies KE.

Hum Mol Genet. 2012 Jun 1;21(11):2559-71. doi: 10.1093/hmg/dds082. Epub 2012 Mar 2.

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