List of works - Sue Fletcher

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

scientific article published on 01 October 2020

A platform for discovery of functional cell-penetrating peptides for efficient multi-cargo intracellular delivery

scientific article published in Scientific Reports

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

scientific article published in July 1997

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

scientific article (publication date: 2 July 2007)

Antisense oligonucleotide induction of progerin in human myogenic cells

scientific article

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

scientific article

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

scientific article published on 18 April 2005

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

scientific article published on 22 May 2019

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

scientific article published on 01 January 2009

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

scientific article published in June 1996

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

scientific article published on 01 January 2009

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

scientific article published on 01 April 2001

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

scientific article published on 8 January 2016

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

scientific article published on 27 January 2017

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

scientific article

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

scientific article

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

scientific article

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

scientific article published on 01 September 2003

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

scientific article published on July 2008

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

scientific article

Gene therapy: therapeutic applications and relevance to pathology

scientific article published on October 2011

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

scientific article

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

scientific article published on April 22, 2013

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

scientific article published on 01 November 2002

Induction of revertant fibres in the mdx mouse using antisense oligonucleotides

scientific article

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

scientific article published on 22 May 2008

Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching

scientific article

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

scientific article published in August 2003

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

scientific article published on 19 June 2007

Multiple exon skipping strategies to by-pass dystrophin mutations ⬇️

scientific article published on December 17, 2011

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

scientific article published on 10 September 2020

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

scientific article published on 18 October 2020

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

scientific article published on 10 March 2011

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

scientific article published on 01 January 2012

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

scientific journal article

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

scientific article published on January 2009

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

scientific article published on December 1, 2010

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

scientific article published on 23 April 2020

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

scientific article published on 31 October 2013

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

scientific article published on 06 August 2020

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

scientific article

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

scientific article published on 01 August 1992

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

scientific article published on 01 August 2011

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

scientific article

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

article

Rational design of antisense oligomers to induce dystrophin exon skipping.

scientific article

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

scientific article published on April 27, 2012

Response to "Railroading at the FDA"

scientific article published on 01 March 2017

Revertant fibers in the mdx murine model of Duchenne muscular dystrophy: an age- and muscle-related reappraisal

scientific article

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

scientific article published on 25 June 2020

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

scientific article

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

scientific article published on 01 January 1998

Specific cloning of DNA fragments unique to the dog Y chromosome

scientific article published on 01 June 1993

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

scientific article

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

scientific article published on January 2010

Splicing intervention for Duchenne muscular dystrophy.

scientific article

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

scientific article

Targeted exon skipping to address "leaky" mutations in the dystrophin gene.

scientific article

Targeted exon skipping to correct exon duplications in the dystrophin gene.

scientific article published on 18 March 2014

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

scientific article published on 04 June 2015

The influence of antisense oligonucleotide length on dystrophin exon skipping.

scientific article

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

scientific article

Translational development of splice-modifying antisense oligomers

scientific article published on 2 November 2016

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

scientific article published in July 1997

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

scientific article published on January 1997

List of works by Sue Fletcher

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

A platform for discovery of functional cell-penetrating peptides for efficient multi-cargo intracellular delivery

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

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

Antisense oligonucleotide induction of progerin in human myogenic cells

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Gene therapy: therapeutic applications and relevance to pathology

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

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

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

Induction of revertant fibres in the mdx mouse using antisense oligonucleotides

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

Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching

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

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

Multiple exon skipping strategies to by-pass dystrophin mutations ⬇️

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Rational design of antisense oligomers to induce dystrophin exon skipping.

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

Response to "Railroading at the FDA"

Revertant fibers in the mdx murine model of Duchenne muscular dystrophy: an age- and muscle-related reappraisal

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

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

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

Specific cloning of DNA fragments unique to the dog Y chromosome

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

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

Splicing intervention for Duchenne muscular dystrophy.

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

Targeted exon skipping to address "leaky" mutations in the dystrophin gene.

Targeted exon skipping to correct exon duplications in the dystrophin gene.

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

The influence of antisense oligonucleotide length on dystrophin exon skipping.

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

Translational development of splice-modifying antisense oligomers

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

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