S. Fletcher

发表

Comprehending the Health Informatics Spectrum: Grappling with System Entropy and Advancing Quality Clinical Research

G. Watts, M. Bellgard, N. Chartres, 2017, Front. Public Health.

Direct dystrophin and reporter gene transfer into dog muscle in vivo

S. Fletcher, J. McC. Howell, A. O'hara, 1998, Muscle & nerve.

Calcium phosphate transfection and cell-specific expression of heterologous genes in primary fetal rat hepatocytes.

S. Fletcher, C. Hilliard, G. Yeoh, 1996, The international journal of biochemistry & cell biology.

Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide

S. Wilton, S. Fletcher, R. Johnsen, 2006, The journal of gene medicine.

Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene

B. Wong, F. Muntoni, R. Kole, 2012, Molecular therapy. Nucleic acids.

Regulation of eukaryotic gene expression by the untranslated gene regions and other non-coding elements

Sue Fletcher, S. Wilton, S. Fletcher, 2012, Cellular and Molecular Life Sciences.

Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy

C. Mann, S. Wilton, S. Fletcher, 2002, The journal of gene medicine.

Gene therapy: therapeutic applications and relevance to pathology

Sue Fletcher, Ian Alexander, John E. J. Rasko, 2011, Pathology.

Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies

S. Wilton, S. Fletcher, A. Adams, 2021, Biomedicines.

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

C. Mann, S. Wilton, S. Fletcher, 2003, Nature Medicine.

Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides

S. Wilton, S. Fletcher, K. Greer, 2019, International journal of molecular sciences.

Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

S. Wilton, S. Fletcher, L. L. Flynn, 2018, Molecular therapy. Nucleic acids.

Induction of revertant fibres in the mdx mouse using antisense oligonucleotides

P. Iversen, S. Wilton, S. Fletcher, 2006, Genetic vaccines and therapy.

Primary over‐expression of AβPP in muscle does not lead to the development of inclusion body myositis in a new lineage of the MCK‐AβPP transgenic mouse

F. Mastaglia, S. Wilton, S. Fletcher, 2013, International journal of experimental pathology.

The Application of Clinical Genetics Dovepress Novel Compounds for the Treatment of Duchenne Muscular Dystrophy: Emerging Therapeutic Agents

S. Wilton, S. Fletcher, 2022 .

Targeted Suppression of a Dystrophin Pseudo-exon using Antisense Oligonucleotides

B. Wong, K. Flanigan, M. Bellgard, 2014 .

Investigation of splicing changes and post-translational processing of LMNA in sporadic inclusion body myositis.

F. Mastaglia, S. Wilton, S. Fletcher, 2013, International journal of clinical and experimental pathology.

Long-term administration of antisense oligonucleotides into the paraspinal muscles of mdx mice reduces kyphosis.

S. Wilton, S. Fletcher, R. Johnsen, 2008, Journal of applied physiology.

Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era

F. Mastaglia, S. Wilton, S. Fletcher, 2020, Medicinal research reviews.

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.

K. Davies, R. Kole, A. Goyenvalle, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Skipping of Duplicated Dystrophin Exons: In Vitro Induction and Assessment.

S. Wilton, S. Fletcher, K. Greer, 2018, Methods in molecular biology.

Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies

B. Wong, G. Pinniger, S. Wilton, 2016, PloS one.

Pseudoexon activation increases phenotype severity in a Becker muscular dystrophy patient

R. Barrero, M. Bellgard, S. Wilton, 2015, Molecular genetics & genomic medicine.

Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching

R. Kole, S. Wilton, S. Fletcher, 2011, BMC Medical Genetics.

High-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscle of dystrophic dogs: prolongation of expression with immunosuppression.

Hanns Lochmüller, J. Nalbantoglu, S. Fletcher, 1998, Human gene therapy.

Dystrophin expression in muscle following gene transfer with a fully deleted ("gutted") adenovirus is markedly improved by trans-acting adenoviral gene products.

J. Nalbantoglu, A. Kamen, S. Fletcher, 2001, Human gene therapy.

RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes.

S. Wilton, S. Fletcher, 2005, Current gene therapy.

Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52

S. Wilton, S. Fletcher, K. Greer, 2021, International journal of molecular sciences.

A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson’s Disease Arising from Parkin Mutations

S. Wilton, S. Fletcher, M. Aung-Htut, 2020, International journal of molecular sciences.

Primary Nasal Epithelial Cells as a Surrogate Cell Culture Model for Type-II Alveolar Cells to Study ABCA-3 Deficiency

S. Stick, A. Kicic, S. Wilton, 2022, Frontiers in Medicine.

Bmc Molecular Biology Antisense Oligonucleotide Induced Exon Skipping and the Dystrophin Gene Transcript: Cocktails and Chemistries

P. Iversen, S. Wilton, S. Fletcher, 2022 .

Induced dystrophin exon skipping in human muscle explants

P. Iversen, H. Moulton, S. Wilton, 2006, Neuromuscular Disorders.

NEAT1 polyA-modulating antisense oligonucleotides reveal opposing functions for both long non-coding RNA isoforms in neuroblastoma

A. Fox, S. Wilton, S. Fletcher, 2020, Cellular and Molecular Life Sciences.

PolyA-modulating antisense oligonucleotides reveal opposing functions for long non-coding RNA NEAT1 isoforms in neuroblastoma

A. Fox, S. Wilton, S. Fletcher, 2020, bioRxiv.

Expanding Patient Access to Cancer Care Services: Key results from a national survey show a range of new initiatives

S. Fletcher, K. D. Simpson, S. Melvin, 2020 .

Antisense-mediated splice intervention to treat human disease: the odyssey continues

S. Wilton, S. Fletcher, L. L. Flynn, 2019, F1000Research.

In Vitro Validation of Phosphorodiamidate Morpholino Oligomers

S. Wilton, S. Fletcher, K. West, 2019, Molecules.

Investigation of age-related changes in LMNA splicing and expression of progerin in human skeletal muscles.

F. Mastaglia, S. Wilton, S. Fletcher, 2013, International journal of clinical and experimental pathology.

Generation of two induced pluripotent stem cell lines from a patient with Stargardt disease caused by compound heterozygous mutations in the ABCA4 gene.

F. Chen, S. Wilton, S. Fletcher, 2021, Stem cell research.

Altered myogenesis and premature senescence underlie human TRIM32-related myopathy

G. Ravenscroft, N. Laing, C. Paradas, 2019, Acta Neuropathologica Communications.

Pax7 includes two polymorphic homeoboxes which contain rearrangements associated with differences in the ability to regenerate damaged skeletal muscle in adult mice.

M. Ziman, S. Fletcher, J. Papadimitriou, 1998, The international journal of biochemistry & cell biology.

Quantitation of muscle precursor cell activity in skeletal muscle by Northern analysis of MyoD and myogenin expression: Application to dystrophic (mdx) mouse muscle

M. Grounds, S. Fletcher, K. Garrett, 1992, Molecular and Cellular Neuroscience.

Cryptic U2-dependent Pre-mRNASplice Site Usage Induced by Splice Switching Antisense Oligonucleotides

S. Wilton, S. Fletcher, K. Greer, 2021 .

Alternate Pax7 transcripts are expressed specifically in skeletal muscle, brain and other organs of adult mice.

M. Ziman, S. Fletcher, P. Kay, 1997, The international journal of biochemistry & cell biology.

The expression of alpha(1)-acid glycoprotein mRNA during rat development. High levels of expression in the decidua.

S. Fletcher, G. Yeoh, G. Schreiber, 1989, The Journal of biological chemistry.

The development of rat α2‐macroglobulin

P. Heinrich, S. Fletcher, G. Yeoh, 1988 .

The emperor's new dystrophin: finding sense in the noise.

S. Wilton, S. Fletcher, R. Veedu, 2015, Trends in molecular medicine.

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

D. A. Stein, B. Neuman, M. Buchmeier, 2007, Biochemical Society transactions.

Splicing intervention for Duchenne muscular dystrophy.

S. Wilton, S. Fletcher, G. Mcclorey, 2005, Current opinion in pharmacology.

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

P. Iversen, H. Moulton, S. Wilton, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle

S. Wilton, S. Fletcher, K. Wells, 2003, FEBS letters.

The influence of antisense oligonucleotide length on dystrophin exon skipping.

S. Wilton, S. Fletcher, K. Honeyman, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Rare disease Roadmap : Navigating the bioinformatics and translational challenges for improved patient health outcomes

C. Mason, M. Sleeman, R. Barrero, 2014 .

Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides

S. Wilton, S. Fletcher, R. Johnsen, 2019, Scientific Reports.

By‐passing the nonsense mutation in the 4CV mouse model of muscular dystrophy by induced exon skipping

P. Iversen, S. Wilton, S. Fletcher, 2009, The journal of gene medicine.

The development of rat alpha 2-macroglobulin. Studies in vivo and in cultured fetal rat hepatocytes.

P. Heinrich, S. Fletcher, G. Yeoh, 1988, European journal of biochemistry.

Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein

S. Wilton, S. Fletcher, R. Johnsen, 2020, Molecular therapy. Nucleic acids.

The spread of transgene expression at the site of gene construct injection

Hanns Lochmüller, S. Fletcher, A. O'hara, 2001, Muscle & nerve.

Pathogenesis and Treatment of Usher Syndrome Type IIA.

F. Chen, S. Wilton, S. Fletcher, 2022, Asia-Pacific journal of ophthalmology.

Evaluation of a short interspersed nucleotide element in the 3' untranslated region of the defective dystrophin gene of dogs with muscular dystrophy.

S. Wilton, S. Fletcher, K. Carville, 2001, American journal of veterinary research.

Use of the dog model for Duchenne muscular dystrophy in gene therapy trials

S. Fletcher, B. Kakulas, H. Lochmuller, 1997, Neuromuscular Disorders.

Translational development of splice-modifying antisense oligomers

M. Bellgard, S. Wilton, S. Fletcher, 2017, Expert opinion on biological therapy.

Proteomic profiling of antisense‐induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm

S. Wilton, S. Fletcher, K. Ohlendieck, 2009, Proteomics.

Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.

S. Wilton, S. Fletcher, A. Fall, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene therapy and molecular approaches to the treatment of hereditary muscular disorders

S. Wilton, S. Fletcher, J. Howell, 2000, Current opinion in neurology.

Modification of pre-mRNA processing: application to dystrophin expression.

S. Wilton, S. Fletcher, 2006, Current opinion in molecular therapeutics.

Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy

F. Muntoni, Haiyan Zhou, S. Wilton, 2013, PloS one.

NEAT1 polyA-modulating antisense oligonucleotides reveal opposing functions for both long non-coding RNA isoforms in neuroblastoma

T. Liu, A. Fox, S. Wilton, 2020, Cellular and Molecular Life Sciences.

Dystrophin as a therapeutic biomarker: Are we ignoring data from the past?

K. Flanigan, S. Wilton, S. Fletcher, 2014, Neuromuscular Disorders.

Gene expression in clonally derived cell lines produced by in vitro transformation of rat fetal hepatocytes: isolation of cell lines which retain liver-specific markers.

S. Fletcher, C. Hilliard, G. Yeoh, 1990, Cancer research.

Personalised genetic intervention for Duchenne muscular dystrophy: antisense oligomers and exon skipping.

S. Wilton, S. Fletcher, C. Mitrpant, 2009, Current molecular pharmacology.

Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro

S. Wilton, S. Fletcher, R. Veedu, 2017, Molecular therapy. Nucleic acids.

Cryptic splicing involving the splice site mutation in the canine model of Duchenne muscular dystrophy

S. Wilton, S. Fletcher, J. Howell, 2001, Neuromuscular Disorders.

Response to “Railroading at the FDA”

F. Muntoni, S. Wilton, S. Fletcher, 2017, Nature Biotechnology.

Consequences of Making the Inactive Active Through Changes in Antisense Oligonucleotide Chemistries

S. Wilton, S. Fletcher, R. Veedu, 2019, Front. Genet..

Analysis of Pathogenic Pseudoexons Reveals Novel Mechanisms Driving Cryptic Splicing

S. Wilton, S. Fletcher, Niall P. Keegan, 2022, Frontiers in Genetics.

Design of a framework for the deployment of collaborative independent rare disease-centric registries: Gaucher disease registry model

N. Zeps, J. Szer, M. Bellgard, 2018, Blood cells, molecules & diseases.

Redirecting splicing to address dystrophin mutations: molecular by-pass surgery.

S. Wilton, S. Fletcher, 2006, Progress in molecular and subcellular biology.

Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.

S. Wilton, S. Fletcher, C. J. Mann, 2003, Human molecular genetics.

Optimizing splice-switching oligomer sequences using 2'-O-methyl phosphorothioate chemistry.

S. Wilton, S. Fletcher, C. Adkin, 2012, Methods in molecular biology.

Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro

Leon M Larcher, C. Bond, A. Fox, 2022, Frontiers in Genetics.

Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid

D. Mackey, F. Chen, S. Wilton, 2017, Vision.

Terminal antisense oligonucleotide modifications can enhance induced exon skipping

S. Wilton, S. Fletcher, R. Johnsen, 2005, Neuromuscular Disorders.

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

P. Iversen, H. Moulton, S. Wilton, 2006, Gene Therapy.

Multiple exon skipping strategies to by-pass dystrophin mutations

B. Wong, F. Muntoni, S. Wilton, 2012, Neuromuscular Disorders.

Untranslated Gene Regions and Other Non-coding Elements: Regulation of Eukaryotic Gene Expression

S. Wilton, S. Fletcher, L. Barrett, 2013 .

Variation in the methylation profile and structure of Pax3 and Pax7 among different mouse strains and during expression.

M. Ziman, S. Fletcher, J. Papadimitriou, 1997, Gene.

Structural Variants May Be a Source of Missing Heritability in sALS

F. Mastaglia, A. Saunders, D. Burns, 2020, Frontiers in Neuroscience.

ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?

S. Wilton, S. Fletcher, L. L. Flynn, 2019, Front. Neurosci..

Exploring microperimetry and autofluorescence endpoints for monitoring disease progression in PRPF31-associated retinopathy

D. Mackey, F. Chen, S. Wilton, 2020, Ophthalmic genetics.

Determinants of Disease Penetrance in PRPF31-Associated Retinopathy

F. Chen, Zhiqin Huang, S. Fletcher, 2021, Genes.

Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene

C. Mann, S. Wilton, S. Fletcher, 2003, The journal of gene medicine.

Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides

S. Agrawal, R. Kole, S. Wilton, 1999, Neuromuscular Disorders.

Splice modulating antisense oligonucleotides restore some acid-alpha-glucosidase activity in cells derived from patients with late-onset Pompe disease

S. Wilton, S. Fletcher, R. Johnsen, 2020, Scientific Reports.

Stargardt disease and progress in therapeutic strategies

F. Chen, S. Wilton, S. Fletcher, 2021, Ophthalmic genetics.

Rare Disease Research Roadmap: Navigating the bioinformatics and translational challenges for improved patient health outcomes

Matthew I. Bellgard, Christopher E. Mason, Alan H. Bittles, 2014 .

YAPping about and not forgetting TAZ

M. Finch-Edmondson, S. Wilton, S. Fletcher, 2019, FEBS letters.

Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now?

S. Wilton, S. Fletcher, 2005, Neuromuscular Disorders.

Antisense Oligonucleotides Targeting Angiogenic Factors as Potential Cancer Therapeutics

S. Wilton, S. Fletcher, R. Veedu, 2018, Molecular therapy. Nucleic acids.

Smart functional nucleic acid chimeras: Enabling tissue specific RNA targeting therapy

S. Wilton, S. Fletcher, R. Veedu, 2015, RNA biology.

Novel Mutations Found in Individuals with Adult-Onset Pompe Disease

S. Wilton, S. Fletcher, M. Aung-Htut, 2020, Genes.

Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene

S. Wilton, S. Fletcher, Y. Nevo, 2020, International journal of molecular sciences.

Breakpoint junction features of seven DMD deletion mutations

S. Wilton, S. Fletcher, Niall P. Keegan, 2019, Human Genome Variation.

Characterization of a complex Duchenne muscular dystrophy–causing dystrophin gene inversion and restoration of the reading frame by induced exon skipping

S. Wilton, S. Fletcher, Mark R. Davis, 2009, Human mutation.

The potential of antisense oligonucleotide therapies for inherited childhood lung diseases

Sue Fletcher, K. Martinovich, S. Stick, 2018, Molecular and Cellular Pediatrics.

Characterising splicing defects of ABCA4 variants within exons 13-50 in patient-derived fibroblasts.

F. Chen, S. Wilton, S. Fletcher, 2022, Experimental eye research.

Generation of an induced pluripotent stem cell line from a patient with Stargardt disease caused by biallelic c.[5461-10T>C;5603A>T];[6077T>C] mutations in the ABCA4 gene.

F. Chen, S. Wilton, S. Fletcher, 2021, Stem cell research.

Phenotype–genotype correlations in a pseudodominant Stargardt disease pedigree due to a novel ABCA4 deletion–insertion variant causing a splicing defect

I. Constable, F. Chen, S. Wilton, 2020, Molecular genetics & genomic medicine.

Antisense oligonucleotide development for the treatment of muscular dystrophies

S. Wilton, S. Fletcher, R. Veedu, 2016 .

Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse.

C. Mann, A. Cheng, S. Wilton, 2001, Proceedings of the National Academy of Sciences of the United States of America.

Induction of cryptic pre-mRNA splice-switching by antisense oligonucleotides

S. Wilton, S. Fletcher, K. Greer, 2021, Scientific Reports.

Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing

S. Wilton, S. Fletcher, M. Aung-Htut, 2019, International journal of molecular sciences.

Antisense suppression of donor splice site mutations in the dystrophin gene transcript

B. Wong, F. Muntoni, S. Wilton, 2013, Molecular genetics & genomic medicine.

A spotter’s guide to SNPtic exons: The common splice variants underlying some SNP–phenotype correlations

S. Fletcher, Niall P. Keegan, 2021, Molecular genetics & genomic medicine.

Nonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1

S. Wilton, S. Fletcher, M. Aung-Htut, 2020, International journal of molecular sciences.

Exon skipping and Duchenne muscular dystrophy: hope, hype and how feasible?

S. Wilton, S. Fletcher, 2008, Neurology India.

Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene

K. Flanigan, Hanns Lochmüller, S. Wilton, 2014, Molecular therapy. Nucleic acids.

Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping.

F. Mastaglia, S. Wilton, S. Fletcher, 2018, Trends in pharmacological sciences.

Antisense Oligonucleotide Induction of Progerin in Human Myogenic Cells

F. Mastaglia, S. Wilton, S. Fletcher, 2014, PloS one.

Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts

Varodom Charoensawan, S. Wilton, S. Fletcher, 2022, International journal of molecular sciences.

Revertant Fibers in the mdx Murine Model of Duchenne Muscular Dystrophy: An Age- and Muscle-Related Reappraisal

C. Romualdi, S. Wilton, S. Fletcher, 2013, PloS one.

Challenges of Interpreting Dystrophin Content by Western Blot

S. Wilton, S. Fletcher, R. Johnsen, 2019, US Neurology.

P.240 Dystrophin expression in the non-DMD population: What is normal?

S. Wilton, S. Fletcher, R. Johnsen, 2016, Neuromuscular Disorders.

Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations

F. Muntoni, Jihee Kim, S. Wilton, 2010, Neuromuscular Disorders.

Molecular diagnosis of duchenne muscular dystrophy: past, present and future in relation to implementing therapies.

N. Laing, S. Wilton, S. Fletcher, 2011, The Clinical biochemist. Reviews.

Polyglutamine ataxias: From Clinical and Molecular Features to Current Therapeutic Strategies

S. Wilton, S. Fletcher, M. Aung-Htut, 2017 .

Altered myogenesis and premature senescence underlie human TRIM32-related myopathy

G. Ravenscroft, N. Laing, C. Paradas, 2019, Acta neuropathologica communications.

Snapback SSCP analysis: Engineered conformation changes for the rapid typing of known mutations

S. Wilton, S. Fletcher, K. Honeyman, 1998, Human mutation.

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

H. Moulton, S. Wilton, S. Fletcher, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics

Leon M Larcher, S. Fletcher, R. Veedu, 2023, Nucleic acid therapeutics.

Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse Model

K. Martinovich, S. Stick, A. Kicic, 2022, Frontiers in Pharmacology.

Novel STMN2 Variant Linked to Amyotrophic Lateral Sclerosis Risk and Clinical Phenotype

I. James, F. Mastaglia, A. Saunders, 2021, Frontiers in Aging Neuroscience.

Gene replacement therapy restores RCBTB1 expression and cilium length in patient‐derived retinal pigment epithelium

F. Chen, Zhiqin Huang, S. Fletcher, 2021, Journal of cellular and molecular medicine.

Interaction of modified oligonucleotides with nuclear proteins, formation of novel nuclear structures and sequence-independent effects on RNA processing

A. Fox, S. Wilton, S. Fletcher, 2018, bioRxiv.

The use of antisense oligonucleotide-mediated exon skipping to treat spinocerebellar ataxia type 3

S. Wilton, S. Fletcher, M. Aung-Htut, 2018 .

Mutation segregation and rapid carrier detection of X-linked muscular dystrophy in dogs.

W. T. Bartlett, R. Bartlett, J. Kornegay, 1996, American journal of veterinary research.

Splice modulating antisense oligonucleotides restore some acid-alpha-glucosidase activity in cells derived from patients with late-onset Pompe disease

S. Wilton, S. Fletcher, R. Johnsen, 2020, Scientific Reports.

Rational design of antisense oligomers to induce dystrophin exon skipping.

F. Muntoni, S. Wilton, S. Fletcher, 2009, Molecular therapy : the journal of the American Society of Gene Therapy.

Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents

S. Wilton, S. Fletcher, 2011 .

Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents.

S. Wilton, S. Fletcher, 2011, The application of clinical genetics.

Splice modification to restore functional dystrophin synthesis in Duchenne muscular dystrophy.

Steve D Wilton, S. Wilton, S. Fletcher, 2010, Current pharmaceutical design.

A Registry Framework Enabling Patient-Centred Care.

Lee Render, Maciej Radochonski, Tarun Weeramanthri, 2015, Studies in health technology and informatics.

Induction of revertant fibres in the mdx Mouse using antisense oligonucleotides

S. Wilton, S. Fletcher, K. Honeyman, 2005 .