about
Smart functional nucleic acid chimeras: enabling tissue specific RNA targeting therapyAntisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistriesPax7 includes two polymorphic homeoboxes which contain rearrangements associated with differences in the ability to regenerate damaged skeletal muscle in adult miceDystrophin as a therapeutic biomarker: are we ignoring data from the past?Proteomic profiling of antisense-induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm.Rational design of antisense oligomers to induce dystrophin exon skipping.Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides.Impaired functional communication between the L-type calcium channel and mitochondria contributes to metabolic inhibition in the mdx heart.Dystrophin isoform induction in vivo by antisense-mediated alternative splicing.Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching.Gene therapy and molecular approaches to the treatment of hereditary muscular disorders.The influence of antisense oligonucleotide length on dystrophin exon skipping.Induction of revertant fibres in the mdx mouse using antisense oligonucleotidesRevertant fibers in the mdx murine model of Duchenne muscular dystrophy: an age- and muscle-related reappraisal.Antisense oligonucleotide induction of progerin in human myogenic cellsDeletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies.Splicing intervention for Duchenne muscular dystrophy.RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes.Targeted exon skipping to address "leaky" mutations in the dystrophin gene.Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agentsExon skipping and Duchenne muscular dystrophy: hope, hype and how feasible?Personalised genetic intervention for Duchenne muscular dystrophy: antisense oligomers and exon skipping.Splice modification to restore functional dystrophin synthesis in Duchenne muscular dystrophy.Targeted exon skipping to correct exon duplications in the dystrophin gene.Gene therapy: therapeutic applications and relevance to pathology.The emperor's new dystrophin: finding sense in the noise.Design of a framework for the deployment of collaborative independent rare disease-centric registries: Gaucher disease registry model.Translational development of splice-modifying antisense oligomers.Dystrophin expression in muscle following gene transfer with a fully deleted ("gutted") adenovirus is markedly improved by trans-acting adenoviral gene products.Variation in the methylation profile and structure of Pax3 and Pax7 among different mouse strains and during expression.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.The spread of transgene expression at the site of gene construct injection.Use of the dog model for Duchenne muscular dystrophy in gene therapy trials.Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene.Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.Alternate Pax7 transcripts are expressed specifically in skeletal muscle, brain and other organs of adult mice.Calcium phosphate transfection and cell-specific expression of heterologous genes in primary fetal rat hepatocytes.Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.A platform for discovery of functional cell-penetrating peptides for efficient multi-cargo intracellular deliveryRare Disease Research Roadmap: Navigating the bioinformatics and translational challenges for improved patient health outcomes
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description
hulumtuese
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researcher
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wetenschapper
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հետազոտող
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name
Sue Fletcher
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Sue Fletcher
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Sue Fletcher
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Sue Fletcher
@nl
Sue Fletcher
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type
label
Sue Fletcher
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Sue Fletcher
@en
Sue Fletcher
@es
Sue Fletcher
@nl
Sue Fletcher
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prefLabel
Sue Fletcher
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Sue Fletcher
@en
Sue Fletcher
@es
Sue Fletcher
@nl
Sue Fletcher
@sl
P106
P1153
7202047963
P21
P31
P496
0000-0002-8632-641X