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Stephen Wilton

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Sebahattin Cirak; George Dickson; Lucy Feng; Maria Elena Garralda; Michela Guglieri; Ryszard Kole; Jennifer E Morgan; Stephen Abbs; Karen Anthony; Virginia Arechavala-Gomeza; John Bourke; Kate Bushby; Caroline Sewry; Stephen B Shrewsbury; Volker Straub; Silvia Torelli; Dominic J Wells; Stephen D Wilton; Matthew J A Wood; Francesco Muntoni
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
Stephen D Wilton; Susan Fletcher
RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes.
Sarah Forrest; Penny L Meloni; Francesco Muntoni; Jihee Kim; Sue Fletcher; Stephen D Wilton
Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations.

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All Publications

2011: Sebahattin Cirak; George Dickson; Lucy Feng; Maria Elena Garralda; Michela Guglieri; Ryszard Kole; Jennifer E Morgan; Stephen Abbs; Karen Anthony; Virginia Arechavala-Gomeza; John Bourke; Kate Bushby; Caroline Sewry; Stephen B Shrewsbury; Volker Straub; Silvia Torelli; Dominic J Wells; Stephen D Wilton; Matthew J A Wood; Francesco Muntoni
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.
Lancet 2011;378(9791):595-605.
2011: Stephen D Wilton; Susan Fletcher
RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes.
Current gene therapy 2011;11(4):259-75.
2010: Sarah Forrest; Penny L Meloni; Francesco Muntoni; Jihee Kim; Sue Fletcher; Stephen D Wilton
Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations.
Neuromuscular disorders : NMD 2010;20(12):810-6.
2010: Sue Fletcher; Abbie M Adams; Russell D Johnsen; Kane Greer; Hong M Moulton; Stephen D Wilton
Dystrophin isoform induction in vivo by antisense-mediated alternative splicing.
Molecular therapy : the journal of the American Society of Gene Therapy 2010;18(6):1218-23.
2009: Chalermchai Mitrpant; Abbie M Adams; Penny L Meloni; Francesco Muntoni; Sue Fletcher; Stephen D Wilton
Rational design of antisense oligomers to induce dystrophin exon skipping.
Molecular therapy : the journal of the American Society of Gene Therapy 2009;17(8):1418-26.
2009: Chalermchai Mitrpant; Sue Fletcher; Patrick L Iversen; Stephen D Wilton
By-passing the nonsense mutation in the 4 CV mouse model of muscular dystrophy by induced exon skipping.
The journal of gene medicine 2009;11(1):46-56.
2009: Heidi R Madden; Sue Fletcher; Mark R Davis; Stephen D Wilton
Characterization of a complex Duchenne muscular dystrophy-causing dystrophin gene inversion and restoration of the reading frame by induced exon skipping.
Human mutation 2009;30(1):22-8.
2008: Stephen D Wilton; Susan Fletcher
Exon skipping and Duchenne muscular dystrophy: hope, hype and how feasible?
Neurology India 2008;56(3):254-62.
2007: Stephen D Wilton
PTC124, nonsense mutations and Duchenne muscular dystrophy.
Neuromuscular disorders : NMD 2007;17(9-10):719-20.
2007: Virginia Arechavala-Gomeza; Ian R Graham; L J Popplewell; A M Adams; Aartsma-Rus Annemieke; Maria Kinali; Jennifer E Morgan; Judith C T van Deutekom; Stephen D Wilton; George Dickson; Francesco Muntoni
Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle.
Human gene therapy 2007;18(9):798-810.
2007: Susan Fletcher; Kaite Honeyman; Abbie M Fall; Penny L Harding; Russell D Johnsen; Joshua P Steinhaus; Hong M Moulton; Patrick L Iversen; Stephen D Wilton
Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(9):1587-92.
2007: Hong M Moulton; Susan Fletcher; Benjamin W Neuman; Graham McClorey; David A Stein; Saïd Abes; Stephen D Wilton; M J Buchmeier; Bernard Lebleu; Patrick L Iversen
Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.
Biochemical Society transactions 2007;35(Pt 4):826-8.
2007: Stephen D Wilton; Abbie M Fall; Penny L Harding; Graham McClorey; Catherine Coleman; Susan Fletcher
Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(7):1288-96.
2007: Bradley T Webb; Edwin J C G van den Oord; Anthony Akkari; Stephen D Wilton; Tina Ly; Rachael Duff; Kathleen C Barnes; Karin Carlsen; Jorrit Gerritsen; Warren Lenney; Michael Silverman; Peter Sly; John Sundy; John Tsanakas; Andrea Von Berg; Moira Whyte; Malcolm Blumenthal; Jørgen Vestbo; Lefkos T Middleton; Peter J Helms; Wayne H Anderson; Sreekumar Pillai
Quantitative linkage genome scan for atopy in a large collection of Caucasian families.
Human genetics 2007;121(1):83-92.
2007: Abbie M Adams; Penny L Harding; Patrick L Iversen; Catherine Coleman; Susan Fletcher; Stephen D Wilton
Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries.
BMC molecular biology 2007;8():57.
2007: Penny L Harding; Abbie M Fall; Kaite Honeyman; Susan Fletcher; Stephen D Wilton
The influence of antisense oligonucleotide length on dystrophin exon skipping.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(1):157-66.
2006: Graham McClorey; Abbie M Fall; Hong M Moulton; Patrick L Iversen; John Rasko; M Ryan; Susan Fletcher; Stephen D Wilton
Induced dystrophin exon skipping in human muscle explants.
Neuromuscular disorders : NMD 2006;16(9-10):583-90.
2006: Graham McClorey; Hong M Moulton; Patrick L Iversen; Susan Fletcher; Stephen D Wilton
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD.
Gene therapy 2006;13(19):1373-81.
2006: Stephen D Wilton; Susan Fletcher
Modification of pre-mRNA processing: application to dystrophin expression.
Current opinion in molecular therapeutics 2006;8(2):130-5.
2006: Julia Alter; Fang Lou; Adam Rabinowitz; HaiFang Yin; Jeffrey Rosenfeld; Stephen D Wilton; Terence A Partridge; Qi-Long Lu
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology.
Nature medicine 2006;12(2):175-7.
2006: Susan Fletcher; Kaite Honeyman; Abbie M Fall; Penny L Harding; Russell D Johnsen; Stephen D Wilton
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide.
The journal of gene medicine 2006;8(2):207-16.
2006: Stephen D Wilton; Susan Fletcher
Redirecting splicing to address dystrophin mutations: molecular by-pass surgery.
Progress in molecular and subcellular biology 2006;44():161-97.
2006: Abbie M Fall; Russell D Johnsen; Kaite Honeyman; Pat Iversen; Susan Fletcher; Stephen D Wilton
Induction of revertant fibres in the mdx mouse using antisense oligonucleotides.
Genetic vaccines and therapy 2006;4():3.
2005: Stephen D Wilton; Susan Fletcher
Antisense oligonucleotides, exon skipping and the dystrophin gene transcript.
Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology / edited by the Gaetano Conte Academy for the study of striated muscle diseases 2005;24(3):222-9.
2005: Stephen D Wilton; Susan Fletcher
RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes.
Current gene therapy 2005;5(5):467-83.
2005: Graham McClorey; Susan Fletcher; Stephen D Wilton
Splicing intervention for Duchenne muscular dystrophy.
Current opinion in pharmacology 2005;5(5):529-34.
2005: Bijanka L Gebski; Stephen J Errington; Russell D Johnsen; Susan Fletcher; Stephen D Wilton
Terminal antisense oligonucleotide modifications can enhance induced exon skipping.
Neuromuscular disorders : NMD 2005;15(9-10):622-9.
2003: Kim E Wells; Susan Fletcher; Christopher J Mann; Stephen D Wilton; Dominic J Wells
Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle.
FEBS letters 2003;552(2-3):145-9.
2003: Bijanka L Gebski; Christopher J Mann; Susan Fletcher; Stephen D Wilton
Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.
Human molecular genetics 2003;12(15):1801-11.
2003: Qi-Long Lu; Christopher J Mann; Fang Lou; George Bou-Gharios; Glenn E Morris; Shao-An Xue; Susan Fletcher; Terence A Partridge; Stephen D Wilton
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.
Nature medicine 2003;9(8):1009-14.
2003: Stephen J Errington; Christopher J Mann; Susan Fletcher; Stephen D Wilton
Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene.
The journal of gene medicine 2003;5(6):518-27.
2002: Christopher J Mann; Kaite Honeyman; Graham McClorey; Susan Fletcher; Stephen D Wilton
Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy.
The journal of gene medicine 2002;4(6):644-54.
2001: Susan Fletcher; Kylie S Carville; JM Howell; Christopher J Mann; Stephen D Wilton
Evaluation of a short interspersed nucleotide element in the 3' untranslated region of the defective dystrophin gene of dogs with muscular dystrophy.
American journal of veterinary research 2001;62(12):1964-8.
2001: Susan Fletcher; T Ly; Rachael M Duff; J McC Howell; Stephen D Wilton
Cryptic splicing involving the splice site mutation in the canine model of Duchenne muscular dystrophy.
Neuromuscular disorders : NMD 2001;11(3):239-43.
2001: Christopher J Mann; Kaite Honeyman; A J Cheng; T Ly; F Lloyd; Susan Fletcher; J E Morgan; Terence A Partridge; Stephen D Wilton
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse.
Proceedings of the National Academy of Sciences of the United States of America 2001;98(1):42-7.
2000: Susan Fletcher; Stephen D Wilton; JM Howell
Gene therapy and molecular approaches to the treatment of hereditary muscular disorders.
Current opinion in neurology 2000;13(5):553-60.
2000: Qi-Long Lu; Glenn E Morris; Stephen D Wilton; T Ly; O V Artem'yeva; P Strong; Terence A Partridge
Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion.
The Journal of cell biology 2000;148(5):985-96.
2000: Kristen J Nowak; P Walsh; Rebecca L Jacob; Russell D Johnsen; J Peverall; Elizabeth McNally; Stephen D Wilton; Byron A Kakulas; Nigel G Laing
Severe gamma-sarcoglycanopathy caused by a novel missense mutation and a large deletion.
Neuromuscular disorders : NMD 2000;10(2):100-7.
1999: P Tan; J Briner; E Boltshauser; Mark R Davis; Stephen D Wilton; Kathryn North; Carina Wallgren-Pettersson; Nigel G Laing
Homozygosity for a nonsense mutation in the alpha-tropomyosin slow gene TPM3 in a patient with severe infantile nemaline myopathy.
Neuromuscular disorders : NMD 1999;9(8):573-9.
1999: Scott J Schatzberg; Natasha Olby; Samantha Steingold; Bruce W Keene; Clarke Atkins; Kathryn M Meurs; G Solomon; S A Goedegebuure; Stephen D Wilton; Nicholas J H Sharp
A polymerase chain reaction screening strategy for the promoter of the canine dystrophin gene.
American journal of veterinary research 1999;60(9):1040-6.
1999: Frank L Mastaglia; Kristen J Nowak; Rick Stell; Bev A Phillips; J E Edmondston; S M Dorosz; Stephen D Wilton; J Hallmayer; Byron A Kakulas; Nigel G Laing
Novel mutation in the myelin protein zero gene in a family with intermediate hereditary motor and sensory neuropathy.
Journal of neurology, neurosurgery, and psychiatry 1999;67(2):174-9.
1999: Stephen D Wilton; F Lloyd; Kylie S Carville; Susan Fletcher; Kaite Honeyman; S Agrawal; Ryszard Kole
Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides.
Neuromuscular disorders : NMD 1999;9(5):330-8.
1999: Scott J Schatzberg; Natasha Olby; Matthew Breen; Louise V B Anderson; Cordelia F Langford; HF Dickens; Stephen D Wilton; C J Zeiss; Matthew M Binns; Joe Kornegay; Glenn E Morris; Nicholas J H Sharp
Molecular analysis of a spontaneous dystrophin 'knockout' dog.
Neuromuscular disorders : NMD 1999;9(5):289-95.
1999: Kaite Honeyman; Kylie S Carville; JM Howell; Susan Fletcher; Stephen D Wilton
Development of a snapback method of single-strand conformation polymorphism analysis for genotyping Golden Retrievers for the X-linked muscular dystrophy allele.
American journal of veterinary research 1999;60(6):734-7.
1999: Peter K Panegyres; Cyril Mamotte; Samuel D Vasikaran; Stephen D Wilton; Vicki Fabian; Byron A Kakulas
Butyrycholinesterase K variant and Alzheimer's disease.
Journal of neurology 1999;246(5):369-70.
1999: Katarina Pelin; P Hilpelä; Kristian Donner; Caroline Sewry; P Anthony Akkari; Stephen D Wilton; Duangrurdee Wattanasirichaigoon; Marie-Louise Bang; T Centner; F Hanefeld; S Odent; M Fardeau; J Andoni Urtizberea; Francesco Muntoni; Victor Dubowitz; Alan H Beggs; Nigel G Laing; Siegfried Labeit; Albert de La Chapelle; Carina Wallgren-Pettersson
Mutations in the nebulin gene associated with autosomal recessive nemaline myopathy.
Proceedings of the National Academy of Sciences of the United States of America 1999;96(5):2305-10.
1998: Scott J Schatzberg; Louise V B Anderson; Stephen D Wilton; Joe Kornegay; Christopher J Mann; G G Solomon; Nicholas J H Sharp
Alternative dystrophin gene transcripts in golden retriever muscular dystrophy.
Muscle & nerve 1998;21(8):991-8.
1998: Stephen D Wilton; Kaite Honeyman; Susan Fletcher; Nigel G Laing
Snapback SSCP analysis: engineered conformation changes for the rapid typing of known mutations.
Human mutation 1998;11(3):252-8.
1997: Katarina Pelin; Maaret Ridanpää; Kristian Donner; Stephen D Wilton; J Krishnarajah; Nigel Laing; B Kolmerer; S Millevoi; Siegfried Labeit; Albert de La Chapelle; C Wallgren-Petterson
Refined localisation of the genes for nebulin and titin on chromosome 2q allows the assignment of nebulin as a candidate gene for autosomal recessive nemaline myopathy.
European journal of human genetics : EJHG 1997;5(4):229-34.
1997: P Tan; J G Allen; Stephen D Wilton; P Anthony Akkari; C R Huxtable; Nigel G Laing
A splice-site mutation causing ovine McArdle's disease.
Neuromuscular disorders : NMD 1997;7(5):336-42.
1997: Stephen D Wilton; Danielle E Dye; Lori Blechynden; Nigel G Laing
Revertant fibres: a possible genetic therapy for Duchenne muscular dystrophy?
Neuromuscular disorders : NMD 1997;7(5):329-35.
1997: Stephen D Wilton; Danielle E Dye; Nigel G Laing
Dystrophin gene transcripts skipping the mdx mutation.
Muscle & nerve 1997;20(6):728-34.
1997: Stephen D Wilton; L Lim; Danielle E Dye; N Laing
Bandstab: a PCR-based alternative to cloning PCR products.
BioTechniques 1997;22(4):642-5.
1996: Stephen D Wilton; L Lim
Long-range PCR: synthesis of products independent of size.
Trends in genetics : TIG 1996;12(11):458.
1996: Betsy A Hosler; G A Nicholson; P C Sapp; W Chin; Richard W Orrell; Jackie de Belleroche; J Esteban; L J Hayward; Diane McKenna-Yasek; L Yeung; A K Cherryson; J E Dench; Stephen D Wilton; Nigel G Laing; H R Horvitz; R H Brown
Three novel mutations and two variants in the gene for Cu/Zn superoxide dismutase in familial amyotrophic lateral sclerosis.
Neuromuscular disorders : NMD 1996;6(5):361-6.
1996: Vicki Fabian; T M Jones; Stephen D Wilton; J E Dench; M R Davis; L Lim; Byron A Kakulas
Alzheimer's disease and apolipoprotein E genotype in Western Australia: an autopsy-verified series.
The Medical journal of Australia 1996;165(2):77-80.
1996: R J Bartlett; N J Winand; S L Secore; J T Singer; S Fletcher; Stephen D Wilton; Daniel J Bogan; Janet Bogan; W T Bartlett; J M Howell; Barry J Cooper; Joe Kornegay
Mutation segregation and rapid carrier detection of X-linked muscular dystrophy in dogs.
American journal of veterinary research 1996;57(5):650-4.
1996: J L Stanton; Stephen D Wilton; Nigel G Laing
Characterisation of the chicken Cu,Zn superoxide dismutase gene.
DNA sequence : the journal of DNA sequencing and mapping 1996;6(6):357-60.
1996: Stephen D Wilton; L Lim; S D Dorosz; H C Gunn; H J Eyre; David F Callen; Nigel G Laing
Assignment of the human alpha-tropomyosin gene TPM4 to band 19p13.1 by fluorescence in situ hybridization.
Cytogenetics and cell genetics 1996;72(4):294-6.
1995: Stephen D Wilton; L Lim
Rapid identification of ApoE alleles by multiple-single-strand conformation polymorphism (SSCP) analysis.
Trends in genetics : TIG 1995;11(9):341.
1995: Nigel G Laing; Stephen D Wilton; P Anthony Akkari; S Dorosz; K Boundy; C Kneebone; P Blumbergs; S White; H Watkins; D R Love
A mutation in the alpha tropomyosin gene TPM3 associated with autosomal dominant nemaline myopathy NEM1.
Nature genetics 1995;10(2):249.
1995: Nigel G Laing; B A Laing; C Meredith; Stephen D Wilton; P Robbins; Kaite Honeyman; S Dorosz; H Kozman; Frank L Mastaglia; Byron A Kakulas
Autosomal dominant distal myopathy: linkage to chromosome 14.
American journal of human genetics 1995;56(2):422-7.
1995: Stephen D Wilton; H Eyre; P Anthony Akkari; H C Watkins; Calum A MacRae; Nigel G Laing; D C Callen
Assignment of the human a-tropomyosin gene TPM3 to 1q22-->q23 by fluorescence in situ hybridisation.
Cytogenetics and cell genetics 1995;68(1-2):122-4.
1995: H Eyre; P Anthony Akkari; Stephen D Wilton; D C Callen; Elizabeth Baker; Nigel G Laing
Assignment of the human skeletal muscle alpha-tropomyosin gene (TPM1) to band 15q22 by fluorescence in situ hybridization.
Cytogenetics and cell genetics 1995;69(1-2):15-7.
1995: Nigel G Laing; Stephen D Wilton; P Anthony Akkari; S Dorosz; K Boundy; C Kneebone; P Blumbergs; S White; H Watkins; D R Love
A mutation in the alpha tropomyosin gene TPM3 associated with autosomal dominant nemaline myopathy.
Nature genetics 1995;9(1):75-9.
1995: C C Hunt; H J Eyre; P Anthony Akkari; C Meredith; S M Dorosz; Stephen D Wilton; David F Callen; Nigel G Laing; Elizabeth Baker
Assignment of the human beta tropomyosin gene (TPM2) to band 9p13 by fluorescence in situ hybridisation.
Cytogenetics and cell genetics 1995;71(1):94-5.
1994: P Anthony Akkari; H J Eyre; Stephen D Wilton; David F Callen; S A Lane; C Meredith; Larry Kedes; Nigel G Laing
Assignment of the human skeletal muscle alpha actin gene (ACTA1) to 1q42 by fluorescence in situ hybridisation.
Cytogenetics and cell genetics 1994;65(4):265-7.
1994: Stephen D Wilton; D C Chandler; Byron A Kakulas; Nigel G Laing
Identification of a point mutation and germinal mosaicism in a Duchenne muscular dystrophy family.
Human mutation 1994;3(2):133-40.
1993: Stephen D Wilton; J Goldblatt; Nigel G Laing
Verification of carrier status for Becker muscular dystrophy from analysis of a blighted ovum.
Prenatal diagnosis 1993;13(8):757-62.
1993: Stephen D Wilton; Russell D Johnsen; J R Pedretti; Nigel G Laing
Two distinct mutations in a single dystrophin gene: identification of an altered splice-site as the primary Becker muscular dystrophy mutation.
American journal of medical genetics 1993;46(5):563-9.
1993: H J Eyre; P Anthony Akkari; C Meredith; Stephen D Wilton; D C Callen; Larry Kedes; Nigel G Laing
Assignment of the human slow skeletal muscle troponin gene (TNNI1) to 1q32 by fluorescence in situ hybridisation.
Cytogenetics and cell genetics 1993;62(2-3):181-2.