All Stories

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

    Article • F1000Research, May 2019, Faculty of 1000, Ltd.

    Professor Sue Fletcher

  2. Response to “Railroading at the FDA”

    Article • Nature Biotechnology, March 2017, Nature

    Professor Sue Fletcher

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

    Article • Blood Cells Molecules and Diseases, January 2017, Elsevier

    Professor Sue Fletcher

  4. Translational development of splice-modifying antisense oligomers

    Article • Expert Opinion on Biological Therapy, November 2016, Taylor & Francis

    Professor Sue Fletcher

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

    Article • PLoS ONE, January 2016, PLOS

    Professor Sue Fletcher

  6. Antisense oligonucleotide development for the treatment of muscular dystrophies

    Article • Expert Opinion on Orphan Drugs, December 2015, Informa Healthcare

    Professor Sue Fletcher

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

    Article • Trends in Molecular Medicine, July 2015, Elsevier

    Professor Sue Fletcher

  8. Pseudoexon activation increases phenotype severity in a Becker muscular dystrophy patient

    Article • Molecular Genetics & Genomic Medicine, April 2015, Wiley

    Professor Sue Fletcher

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

    Article • RNA Biology, April 2015, Taylor & Francis

    Professor Sue Fletcher

  10. Morpholino Oligomer Peptide Therapy Improves Mitochondrial Function in mdx Cardiomyopathy

    Article • Biophysical Journal, January 2015, Elsevier

    Professor Sue Fletcher

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

    Article • Health Policy and Technology, December 2014, Elsevier

    Professor Sue Fletcher

  12. Impaired functional communication between the L-type calcium channel and mitochondria contributes to metabolic inhibition in the mdx heart

    Article • Proceedings of the National Academy of Sciences, June 2014, Proceedings of the National Academy of Sciences

    Professor Sue Fletcher

  13. Antisense Oligonucleotide Induction of Progerin in Human Myogenic Cells

    Article • PLoS ONE, June 2014, PLOS

    Professor Sue Fletcher

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

    Article • Neuromuscular Disorders, June 2014, Elsevier

    Professor Sue Fletcher

  15. O10 Development of PMO antisense oligonucleotides for treatment of Spinal muscular atrophy

    Article • Neuromuscular Disorders, March 2014, Elsevier

    Professor Sue Fletcher

  16. Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene

    Article • Molecular Therapy — Nucleic Acids, January 2014, Elsevier

    Professor Sue Fletcher

  17. Primary over-expression of AβPP in muscle does not lead to the development of inclusion body myositis in a new lineage of theMCK-AβPPtransgenic mouse

    Article • International Journal of Experimental Pathology, October 2013, Wiley

    Professor Sue Fletcher

  18. P.6.5 Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing

    Article • Neuromuscular Disorders, October 2013, Elsevier

    Professor Sue Fletcher

  19. P.6.6 Splice switching Antisense Oligonucleotides: A “kink” in the design?

    Article • Neuromuscular Disorders, October 2013, Elsevier

    Professor Sue Fletcher

  20. P.11.5 PMO-mediated dystrophin exon 23 skipping restores mitochondrial function in the mdx mouse heart

    Article • Neuromuscular Disorders, October 2013, Elsevier

    Professor Sue Fletcher

  21. P.19.2 Targeting the Huntingtin gene with splice-switching AOs: An unusual result

    Article • Neuromuscular Disorders, October 2013, Elsevier

    Professor Sue Fletcher

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

    Article • PLoS ONE, August 2013, PLOS

    Professor Sue Fletcher

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

    Article • Molecular Genetics & Genomic Medicine, June 2013, Wiley

    Professor Sue Fletcher

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

    Article • PLoS ONE, April 2013, PLOS

    Professor Sue Fletcher

  25. Untranslated Gene Regions and Other Non-coding Elements

    Article • January 2013

    Professor Sue Fletcher

  26. Untranslated Gene Regions and Other Non-coding Elements

    Article • January 2013, Springer Science + Business Media

    Professor Sue Fletcher

  27. G.P.78 Dystrophin isoforms with incomplete β dystroglycan and syntrophin binding domains retain partial function

    Article • Neuromuscular Disorders, October 2012, Elsevier

    Professor Sue Fletcher

  28. T.P.42 Antisense oligonucleotide induced over-expression of progerin in human myoblasts: A possible model of muscle aging

    Article • Neuromuscular Disorders, October 2012, Elsevier

    Professor Sue Fletcher

  29. G.P.64 Histological, immunohistochemical and electron microscopic study of muscle in MCK-βAPP mice

    Article • Neuromuscular Disorders, October 2012, Elsevier

    Professor Sue Fletcher

  30. T.P.19 Enhanced exon skipping in the 4CV dystrophic mouse model of muscular dystrophy through refined oligomer design

    Article • Neuromuscular Disorders, October 2012, Elsevier

    Professor Sue Fletcher

  31. T.P.28 Pseudo-exon inactivation of the dystrophin gene: Ideal candidates for exon skipping

    Article • Neuromuscular Disorders, October 2012, Elsevier

    Professor Sue Fletcher

  32. T.P.30 Characterisation of two DMD-causing splice-site mutations and development of personalised exon skipping strategies

    Article • Neuromuscular Disorders, October 2012, Elsevier

    Professor Sue Fletcher

  33. T.P.32 Developing a transient mouse model of Spinal Muscular Atrophy to determine the functionality of SMN on peripheral tissue

    Article • Neuromuscular Disorders, October 2012, Elsevier

    Professor Sue Fletcher

  34. T.P.33 A cutting-edge approach to Spinal Muscular Atrophy treatment using antisense oligonucleotides

    Article • Neuromuscular Disorders, October 2012, Elsevier

    Professor Sue Fletcher

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

    Article • Cellular and Molecular Life Sciences, April 2012, Springer Science + Business Media

    Professor Sue Fletcher

  36. Multiple exon skipping strategies to by-pass dystrophin mutations

    Article • Neuromuscular Disorders, April 2012, Elsevier

    Professor Sue Fletcher

  37. Optimizing Splice-Switching Oligomer Sequences Using 2′-O-Methyl Phosphorothioate Chemistry

    Article • January 2012, Springer Science + Business Media

    Professor Sue Fletcher

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

    Article • Molecular Therapy — Nucleic Acids, January 2012, Elsevier

    Professor Sue Fletcher

  39. Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching

    Article • BMC Medical Genetics, October 2011, Springer Science + Business Media

    Professor Sue Fletcher

  40. Gene therapy: therapeutic applications and relevance to pathology

    Article • Pathology, October 2011, Elsevier

    Professor Sue Fletcher

  41. P4.31 Transient mouse models for the preclinical evaluation of therapeutic dystrophin exon skipping strategies

    Article • Neuromuscular Disorders, October 2011, Elsevier

    Professor Sue Fletcher

  42. P4.30 Multiple exon skipping strategies to by-pass selected dystrophin mutations

    Article • Neuromuscular Disorders, October 2011, Elsevier

    Professor Sue Fletcher

  43. P5.34 Alternative splicing of lamin A leads to age-dependent accumulation of progerin transcript in normal human muscle and sporadic IBM

    Article • Neuromuscular Disorders, October 2011, Elsevier

    Professor Sue Fletcher

  44. P3.11 Antisense oligomer mediated retention of SMN2 intron and exon 7 leads to a novel SMN transcript and increased functional SMN protein in Spinal Muscular Atrophy fibroblasts

    Article • Neuromuscular Disorders, October 2011, Elsevier

    Professor Sue Fletcher

  45. RNA Splicing Manipulation: Strategies to Modify Gene Expression for a Variety of Therapeutic Outcomes

    Article • Current Gene Therapy, August 2011, Bentham Science Publishers

    Professor Sue Fletcher

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

    Article • The Application of Clinical Genetics, March 2011, Taylor & Francis

    Professor Sue Fletcher

  47. P18 Transient mouse models for the preclinical evaluation of therapeutic dystrophin exon skipping strategies

    Article • Neuromuscular Disorders, March 2011, Elsevier

    Professor Sue Fletcher

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

    Article • Neuromuscular Disorders, December 2010, Elsevier

    Professor Sue Fletcher

  49. P3.32 Exon/intron 7 inclusion: an alternative mode of splice intervention in spinal muscular atrophy fibroblasts

    Article • Neuromuscular Disorders, October 2010, Elsevier

    Professor Sue Fletcher

  50. P3.12 Concurrent administration of prednisolone and peptide conjugated PMOs is not contraindicated in the mdx mouse

    Article • Neuromuscular Disorders, October 2010, Elsevier

    Professor Sue Fletcher

  51. Dystrophin Isoform Induction In Vivo by Antisense-mediated Alternative Splicing

    Article • Molecular Therapy, June 2010, Elsevier

    Professor Sue Fletcher

  52. Splice Modification to Restore Functional Dystrophin Synthesis in Duchenne Muscular Dystrophy

    Article • Current Pharmaceutical Design, March 2010, Bentham Science Publishers

    Professor Sue Fletcher

  53. Splice Modification to Restore Functional Dystrophin Synthesis in Duchenne Muscular Dystrophy

    Article • Current Pharmaceutical Design, March 2010, Bentham Science Publishers

    Professor Sue Fletcher

  54. P26 Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping

    Article • Neuromuscular Disorders, March 2010, Elsevier

    Professor Sue Fletcher

  55. Splice-switching as a treament for duchenne muscular dystrophy

    Article • Pathology, January 2010, Elsevier

    Professor Sue Fletcher

  56. Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

    Article • Molecular Therapy, January 2010, Elsevier

    Professor Sue Fletcher

  57. T.P.1.06 Induced non-productive splicing to study muscle gene expression

    Article • Neuromuscular Disorders, September 2009, Elsevier

    Professor Sue Fletcher

  58. T.P.1.08 The use of antisense oligomer for splice switching in spinal muscular atrophy fibroblasts

    Article • Neuromuscular Disorders, September 2009, Elsevier

    Professor Sue Fletcher

  59. Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping

    Article • Molecular Therapy, August 2009, Elsevier

    Professor Sue Fletcher

  60. Proteomic profiling of antisense-induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm

    Article • PROTEOMICS, February 2009, Wiley

    Professor Sue Fletcher

  61. Personalised Genetic Intervention for Duchenne Muscular Dystrophy: Antisense Oligomers and Exon Skipping

    Article • Current Molecular Pharmacology, January 2009, Bentham Science Publishers

    Professor Sue Fletcher

  62. Personalised Genetic Intervention for Duchenne Muscular Dystrophy: Antisense Oligomers and Exon Skipping

    Article • Current Molecular Pharmacology, January 2009, Bentham Science Publishers

    Professor Sue Fletcher

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

    Article • Human Mutation, January 2009, Wiley

    Professor Sue Fletcher

  64. By-passing the nonsense mutation in the 4CVmouse model of muscular dystrophy by induced exon skipping

    Article • The Journal of Gene Medicine, January 2009, Wiley

    Professor Sue Fletcher

  65. T.I.4 Splice manipulation therapies: Opportunities and challenges

    Article • Neuromuscular Disorders, October 2008, Elsevier

    Professor Sue Fletcher

  66. T.P.2.01 Antisense oligomer design: Targeting and assay systems

    Article • Neuromuscular Disorders, October 2008, Elsevier

    Professor Sue Fletcher

  67. T.P.2.03 Characterisation of a complex dystrophin mutation: Assume nothing when designing exon skipping strategies

    Article • Neuromuscular Disorders, October 2008, Elsevier

    Professor Sue Fletcher

  68. T.P.2.09 Induced exon skipping in normal and mdx muscle

    Article • Neuromuscular Disorders, October 2008, Elsevier

    Professor Sue Fletcher

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

    Article • Journal of Applied Physiology, May 2008, American Physiological Society

    Professor Sue Fletcher

  70. Exon skipping and Duchenne muscular dystrophy: Hope, hype and how feasible?

    Article • Neurology India, January 2008, Medknow

    Professor Sue Fletcher

  71. Morpholino Oligomer–Mediated Exon Skipping Averts the Onset of Dystrophic Pathology in the mdx Mouse

    Article • Molecular Therapy, September 2007, Elsevier

    Professor Sue Fletcher

  72. Cell-penetrating peptide–morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo : Figure 1

    Article • Biochemical Society Transactions, August 2007, Portland Press Ltd.

    Benjamin W Neuman, Professor Sue Fletcher

  73. Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene Transcript

    Article • Molecular Therapy, July 2007, Elsevier

    Professor Sue Fletcher

  74. Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries

    Article • BMC Molecular Biology, January 2007, Springer Science + Business Media

    Professor Sue Fletcher

  75. The Influence of Antisense Oligonucleotide Length on Dystrophin Exon Skipping

    Article • Molecular Therapy, January 2007, Elsevier

    Professor Sue Fletcher

  76. Induced dystrophin exon skipping in human muscle explants

    Article • Neuromuscular Disorders, October 2006, Elsevier

    Professor Sue Fletcher

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

    Article • Gene Therapy, May 2006, Springer Science + Business Media

    Professor Sue Fletcher

  78. Redirecting Splicing to Address Dystrophin Mutations: Molecular By-pass Surgery

    Article • January 2006, Springer Science + Business Media

    Professor Sue Fletcher

  79. Dystrophin expression in themdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide

    Article • The Journal of Gene Medicine, January 2006, Wiley

    Professor Sue Fletcher

  80. Article • Genetic Vaccines and Therapy, January 2006, Springer Science + Business Media

    Professor Sue Fletcher

  81. Splicing intervention for Duchenne muscular dystrophy

    Article • Current Opinion in Pharmacology, October 2005, Elsevier

    Professor Sue Fletcher

  82. RNA Splicing Manipulation: Strategies to Modify Gene Expression for a Variety of Therapeutic Outcomes

    Article • Current Gene Therapy, October 2005, Bentham Science Publishers

    Professor Sue Fletcher

  83. Terminal antisense oligonucleotide modifications can enhance induced exon skipping

    Article • Neuromuscular Disorders, October 2005, Elsevier

    Professor Sue Fletcher

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

    Article • Neuromuscular Disorders, June 2005, Elsevier

    Professor Sue Fletcher

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

    Article • FEBS Letters, August 2003, Wiley

    Professor Sue Fletcher

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

    Article • Human Molecular Genetics, July 2003, Oxford University Press (OUP)

    Professor Sue Fletcher

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

    Article • Nature Reports Stem Cells, July 2003, Springer Science + Business Media

    Professor Sue Fletcher

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

    Article • The Journal of Gene Medicine, January 2003, Wiley

    Professor Sue Fletcher

  89. Improved antisense oligonucleotide induced exon skipping in themdx mouse model of muscular dystrophy

    Article • The Journal of Gene Medicine, January 2002, Wiley

    Professor Sue Fletcher

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

    Article • American Journal of Veterinary Research, December 2001, American Veterinary Medical Association (AVMA)

    Professor Sue Fletcher

  91. Dystrophin Expression in Muscle Following Gene Transfer with a Fully Deleted ("Gutted") Adenovirus Is Markedly Improved by Trans-Acting Adenoviral Gene Products

    Article • Human Gene Therapy, September 2001, Mary Ann Liebert Inc

    Professor Sue Fletcher

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

    Article • Neuromuscular Disorders, April 2001, Elsevier

    Professor Sue Fletcher

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

    Article • Muscle & Nerve, March 2001, Wiley

    Professor Sue Fletcher

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

    Article • Proceedings of the National Academy of Sciences, December 2000, Proceedings of the National Academy of Sciences

    Professor Sue Fletcher

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

    Article • Proceedings of the National Academy of Sciences, December 2000, Proceedings of the National Academy of Sciences

    Professor Sue Fletcher

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

    Article • Current Opinion in Neurology, October 2000, Wolters Kluwer Health

    Professor Sue Fletcher

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

    Article • Neuromuscular Disorders, July 1999, Elsevier

    Professor Sue Fletcher

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

    Article • The International Journal of Biochemistry & Cell Biology, March 1998, Elsevier

    Professor Sue Fletcher

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

    Article • Human Mutation, January 1998, Wiley

    Professor Sue Fletcher

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

    Article • Human Mutation, January 1998, Wiley

    Professor Sue Fletcher

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

    Article • The International Journal of Biochemistry & Cell Biology, July 1997, Elsevier

    Professor Sue Fletcher

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

    Article • Neuromuscular Disorders, July 1997, Elsevier

    Professor Sue Fletcher

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

    Article • Gene, January 1997, Elsevier

    Professor Sue Fletcher

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

    Article • The International Journal of Biochemistry & Cell Biology, June 1996, Elsevier

    Professor Sue Fletcher

  105. Specific cloning of DNA fragments unique to the dog Y chromosome

    Article • Genetic Analysis Biomolecular Engineering, June 1993, Elsevier

    Professor Sue Fletcher

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

    Article • Molecular and Cellular Neuroscience, August 1992, Elsevier

    Professor Sue Fletcher

  107. The development of rat alpha2-macroglobulin. Studies in vivo and in cultured fetal rat hepatocytes

    Article • European Journal of Biochemistry, February 1988, Wiley

    Professor Sue Fletcher