Sue Fletcher

Q: What Schools Are Affiliated With Sue Fletcher

Sue Fletcher is affiliated with the following schools: University of Melbourne, University of Zimbabwe, Murdoch University, Australian National University, University of Western Australia

Sue Fletcher's AcademicInfluence.com Rankings

Sue Fletcher

Chemistry

#5416

World Rank

#6588

Historical Rank

Organic Chemistry

#966

World Rank

#1090

Historical Rank

chemistry Degrees

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Chemistry

Sue Fletcher's Degrees

Bachelors Chemistry University of Melbourne

Why Is Sue Fletcher Influential?

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According to Wikipedia, Sue Fletcher is a professor and lecturer at the University of Western Australia and a Senior Principal Research Fellow at Murdoch University. She studies the field of molecular and cell biology and holds the position of chief scientific officer at PYC therapeutics. Currently her research is focused on inherited retinal disease, the central nervous system and neurodegeneration. Alongside Professor Steve Winton she developed novel treatments for Duchenne muscular dystrophy, including eteplirsen, which was the first of its kind to be approved by the FDA in 2016. In 2021 she was appointed an Officer of the Order of Australia for "distinguished service to medical research, to neurological science, and to the treatment and support of those with Muscular Dystrophy".

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Sue Fletcher's Published Works

Number of citations in a given year to any of this author's works

Total number of citations to an author for the works they published in a given year. This highlights publication of the most important work(s) by the author

Published Works

Regulation of eukaryotic gene expression by the untranslated gene regions and other non-coding elements (2012) (480)
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse (2003) (405)
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. (2001) (385)
ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now? (2019) (354)
Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides (1999) (224)
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD (2006) (215)
Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. (2003) (189)
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide (2006) (181)
Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse. (2007) (165)
Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript. (2007) (162)
Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy (2002) (158)
Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping. (2010) (105)
High-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscle of dystrophic dogs: prolongation of expression with immunosuppression. (1998) (90)
Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo. (2007) (82)
The influence of antisense oligonucleotide length on dystrophin exon skipping. (2007) (79)
Use of the dog model for Duchenne muscular dystrophy in gene therapy trials (1997) (74)
Induced dystrophin exon skipping in human muscle explants (2006) (74)
Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries (2007) (73)
Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy (2013) (71)
Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle (2003) (63)
Proteomic profiling of antisense‐induced exon skipping reveals reversal of pathobiochemical abnormalities in dystrophic mdx diaphragm (2009) (60)
Dystrophin expression in muscle following gene transfer with a fully deleted ("gutted") adenovirus is markedly improved by trans-acting adenoviral gene products. (2001) (60)
Mutation segregation and rapid carrier detection of X-linked muscular dystrophy in dogs. (1996) (55)
Quantitation of muscle precursor cell activity in skeletal muscle by Northern analysis of MyoD and myogenin expression: Application to dystrophic (mdx) mouse muscle (1992) (54)
RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes. (2005) (51)
Antisense Oligonucleotides Targeting Angiogenic Factors as Potential Cancer Therapeutics (2018) (48)
By‐passing the nonsense mutation in the 4CV mouse model of muscular dystrophy by induced exon skipping (2009) (47)
Induction of revertant fibres in the mdx mouse using antisense oligonucleotides (2006) (45)
Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene (2003) (43)
Rational design of antisense oligomers to induce dystrophin exon skipping. (2009) (42)
The emperor's new dystrophin: finding sense in the noise. (2015) (42)
Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping. (2018) (41)
Molecular diagnosis of duchenne muscular dystrophy: past, present and future in relation to implementing therapies. (2011) (39)
The spread of transgene expression at the site of gene construct injection (2001) (39)
Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene (2014) (38)
Alternate Pax7 transcripts are expressed specifically in skeletal muscle, brain and other organs of adult mice. (1997) (38)
Characterization of a complex Duchenne muscular dystrophy–causing dystrophin gene inversion and restoration of the reading frame by induced exon skipping (2009) (38)
Novel STMN2 Variant Linked to Amyotrophic Lateral Sclerosis Risk and Clinical Phenotype (2021) (33)
Smart functional nucleic acid chimeras: Enabling tissue specific RNA targeting therapy (2015) (31)
Development of a snapback method of single-strand conformation polymorphism analysis for genotyping Golden Retrievers for the X-linked muscular dystrophy allele. (1999) (31)
YAPping about and not forgetting TAZ (2019) (30)
Structural Variants May Be a Source of Missing Heritability in sALS (2020) (30)
Exon skipping and Duchenne muscular dystrophy: hope, hype and how feasible? (2008) (30)
The expression of alpha(1)-acid glycoprotein mRNA during rat development. High levels of expression in the decidua. (1989) (29)
Revertant Fibers in the mdx Murine Model of Duchenne Muscular Dystrophy: An Age- and Muscle-Related Reappraisal (2013) (27)
Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro (2017) (26)
The development of rat α2‐macroglobulin (1988) (26)
NEAT1 polyA-modulating antisense oligonucleotides reveal opposing functions for both long non-coding RNA isoforms in neuroblastoma (2020) (25)
Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies (2016) (24)
Splicing intervention for Duchenne muscular dystrophy. (2005) (23)
Dystrophin isoform induction in vivo by antisense-mediated alternative splicing. (2010) (23)
Personalised genetic intervention for Duchenne muscular dystrophy: antisense oligomers and exon skipping. (2009) (22)
The potential of antisense oligonucleotide therapies for inherited childhood lung diseases (2018) (22)
Pseudoexon activation increases phenotype severity in a Becker muscular dystrophy patient (2015) (22)
Terminal antisense oligonucleotide modifications can enhance induced exon skipping (2005) (22)
Altered myogenesis and premature senescence underlie human TRIM32-related myopathy (2019) (22)
Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene (2012) (21)
Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era (2020) (20)
Translational development of splice-modifying antisense oligomers (2017) (19)
Gene therapy and molecular approaches to the treatment of hereditary muscular disorders (2000) (19)
Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now? (2005) (18)
Untranslated Gene Regions and Other Non-coding Elements: Regulation of Eukaryotic Gene Expression (2013) (18)
Modification of pre-mRNA processing: application to dystrophin expression. (2006) (18)
Dystrophin as a therapeutic biomarker: Are we ignoring data from the past? (2014) (17)
Multiple exon skipping strategies to by-pass dystrophin mutations (2012) (17)
Antisense oligonucleotide development for the treatment of muscular dystrophies (2016) (17)
Snapback SSCP analysis: Engineered conformation changes for the rapid typing of known mutations (1998) (17)
Variation in the methylation profile and structure of Pax3 and Pax7 among different mouse strains and during expression. (1997) (17)
Design of a framework for the deployment of collaborative independent rare disease-centric registries: Gaucher disease registry model (2018) (16)
Splice modification to restore functional dystrophin synthesis in Duchenne muscular dystrophy. (2010) (16)
Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations (2010) (15)
Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides (2019) (14)
Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript (2018) (14)
Direct dystrophin and reporter gene transfer into dog muscle in vivo (1998) (14)
Gene therapy: therapeutic applications and relevance to pathology (2011) (13)
The development of rat alpha 2-macroglobulin. Studies in vivo and in cultured fetal rat hepatocytes. (1988) (13)
A Registry Framework Enabling Patient-Centred Care. (2015) (13)
In Vitro Validation of Phosphorodiamidate Morpholino Oligomers (2019) (13)
Antisense suppression of donor splice site mutations in the dystrophin gene transcript (2013) (12)
Rare Disease Research Roadmap: Navigating the bioinformatics and translational challenges for improved patient health outcomes (2014) (12)
Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides (2019) (12)
Antisense Oligonucleotide Induction of Progerin in Human Myogenic Cells (2014) (12)
Cryptic splicing involving the splice site mutation in the canine model of Duchenne muscular dystrophy (2001) (11)
Investigation of age-related changes in LMNA splicing and expression of progerin in human skeletal muscles. (2013) (11)
Long-term administration of antisense oligonucleotides into the paraspinal muscles of mdx mice reduces kyphosis. (2008) (11)
Phenotype–genotype correlations in a pseudodominant Stargardt disease pedigree due to a novel ABCA4 deletion–insertion variant causing a splicing defect (2020) (10)
Removal of the Polyglutamine Repeat of Ataxin-3 by Redirecting pre-mRNA Processing (2019) (9)
Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching (2011) (9)
Nonsequential Splicing Events Alter Antisense-Mediated Exon Skipping Outcome in COL7A1 (2020) (9)
Antisense oligonucleotides, exon skipping and the dystrophin gene transcript. (2005) (8)
Pax7 includes two polymorphic homeoboxes which contain rearrangements associated with differences in the ability to regenerate damaged skeletal muscle in adult mice. (1998) (8)
Antisense-mediated splice intervention to treat human disease: the odyssey continues (2019) (8)
Stargardt disease and progress in therapeutic strategies (2021) (8)
Polyglutamine ataxias: From Clinical and Molecular Features to Current Therapeutic Strategies (2017) (7)
Redirecting splicing to address dystrophin mutations: molecular by-pass surgery. (2006) (7)
Generation of two induced pluripotent stem cell lines from a patient with dominant PRPF31 mutation and a related non-penetrant carrier. (2019) (7)
A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson’s Disease Arising from Parkin Mutations (2020) (7)
Investigation of splicing changes and post-translational processing of LMNA in sporadic inclusion body myositis. (2013) (7)
Response to “Railroading at the FDA” (2017) (7)
Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid (2017) (7)
Determinants of Disease Penetrance in PRPF31-Associated Retinopathy (2021) (6)
Calcium phosphate transfection and cell-specific expression of heterologous genes in primary fetal rat hepatocytes. (1996) (6)
Interaction of modified oligonucleotides with nuclear proteins, formation of novel nuclear structures and sequence-independent effects on RNA processing (2018) (6)
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. (1990) (6)
Breakpoint junction features of seven DMD deletion mutations (2019) (6)
Exploring microperimetry and autofluorescence endpoints for monitoring disease progression in PRPF31-associated retinopathy (2020) (6)
Challenges of Interpreting Dystrophin Content by Western Blot (2019) (6)
Novel Mutations Found in Individuals with Adult-Onset Pompe Disease (2020) (6)
Analysis of Pathogenic Pseudoexons Reveals Novel Mechanisms Driving Cryptic Splicing (2022) (6)
Splice modulating antisense oligonucleotides restore some acid-alpha-glucosidase activity in cells derived from patients with late-onset Pompe disease (2020) (5)
Evaluation of a short interspersed nucleotide element in the 3' untranslated region of the defective dystrophin gene of dogs with muscular dystrophy. (2001) (5)
Exon skipping prevents the onset of dystrophic pathology in the MDX mouse (2007) (4)
Consequences of Making the Inactive Active Through Changes in Antisense Oligonucleotide Chemistries (2019) (4)
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 (2013) (4)
Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents (2011) (4)
RNA splicing manipulation: strategies to modify gene expression for a variety of therapeutic outcomes. (2011) (3)
Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein (2020) (3)
Optimizing splice-switching oligomer sequences using 2'-O-methyl phosphorothioate chemistry. (2012) (3)
Morpholino antisense oligonucleotide induced dystrophin expression in the MDX mouse (2006) (3)
Targeted Suppression of a Dystrophin Pseudo-exon using Antisense Oligonucleotides (2014) (3)
Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies (2021) (3)
D35 Development of a validated Western blot method for quantification of human dystrophin protein used in phase 2 and 3 clinical trials of eteplirsen for the treatment of Duchenne muscular dystrophy (2017) (2)
Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro (2022) (2)
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. (2021) (2)
Generation of three induced pluripotent stem cell lines from a patient with Usher syndrome caused by biallelic c.949C > A and c.1256G > T mutations in the USH2A gene. (2020) (2)
Single Exon Skipping Can Address a Multi-Exon Duplication in the Dystrophin Gene (2020) (2)
Comprehending the Health Informatics Spectrum: Grappling with System Entropy and Advancing Quality Clinical Research (2017) (2)
P5.34 Alternative splicing of lamin A leads to age-dependent accumulation of progerin transcript in normal human muscle and sporadic IBM (2011) (2)
Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse Model (2022) (1)
Expanding Patient Access to Cancer Care Services: Key results from a national survey show a range of new initiatives (2020) (1)
T.I.4 Splice manipulation therapies: Opportunities and challenges (2008) (1)
Targeted dystrophin gene processing using antisense oligonucleotides (1998) (1)
The use of antisense oligonucleotide-mediated exon skipping to treat spinocerebellar ataxia type 3 (2018) (1)
Morpholino antisense oligonucleotides for inducing exon skipping (2003) (1)
Proof-of-Concept: Antisense Oligonucleotide Mediated Skipping of Fibrillin-1 Exon 52 (2021) (1)
Faculty Opinions recommendation of Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index. (2019) (1)
P.240 Dystrophin expression in the non-DMD population: What is normal? (2016) (1)
Rare disease Roadmap : Navigating the bioinformatics and translational challenges for improved patient health outcomes (2014) (1)
A spotter’s guide to SNPtic exons: The common splice variants underlying some SNP–phenotype correlations (2021) (1)
PolyA-modulating antisense oligonucleotides reveal opposing functions for long non-coding RNA NEAT1 isoforms in neuroblastoma (2020) (1)
Pathogenesis and Treatment of Usher Syndrome Type IIA. (2022) (1)
Skipping of Duplicated Dystrophin Exons: In Vitro Induction and Assessment. (2018) (1)
Induction of cryptic pre-mRNA splice-switching by antisense oligonucleotides (2021) (1)
Gene replacement therapy restores RCBTB1 expression and cilium length in patient‐derived retinal pigment epithelium (2021) (1)
Gene therapy in the dog model for Duchenne muscular dystrophy (1997) (0)
Rescue of CFTR function impaired by mutations in exon 15 (2020) (0)
T.P.19 Enhanced exon skipping in the 4CV dystrophic mouse model of muscular dystrophy through refined oligomer design (2012) (0)
Generation of two induced pluripotent stem cell lines from a patient with Stargardt disease caused by compound heterozygous mutations in the ABCA4 gene. (2021) (0)
Concurrent administration of prednisolone and peptide conjugated PMOs is not contra-indicated in the MDX mouse (2010) (0)
Faculty Opinions recommendation of Injection site reactions after long-term subcutaneous delivery of drisapersen: a retrospective study. (2019) (0)
Treatment of mdx Mice with Antisense Oligomers to Induce Exon Skipping of Dystrophin Exon 23 Restores Mitochondrial Function in mdx Cardiomyopathy (2013) (0)
Faculty Opinions recommendation of A Sensitive In Vitro Approach to Assess the Hybridization-Dependent Toxic Potential of High Affinity Gapmer Oligonucleotides. (2019) (0)
Antisense induced redirection of splicing: Will duchenne muscular dystrophy be the thin edge of the wedge? (2005) (0)
Sequence of full-length dystrophin CDNA and several revertant fibres from muscles of golden retriever dogs (1997) (0)
PMO-mediated dystrophin exon 23 skipping restores nitochondrial function in the MDX heart (2013) (0)
Exceptions to the reading frame rule in DMD: Will exon skipping be relevant? (2009) (0)
Faculty Opinions recommendation of Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History. (2019) (0)
A clinical trial update on Exon skipping and Duchenne muscular dystrophy (2014) (0)
P3.32 Exon/intron 7 inclusion: an alternative mode of splice intervention in spinal muscular atrophy fibroblasts (2010) (0)
P3.12 Concurrent administration of prednisolone and peptide conjugated PMOs is not contraindicated in the mdx mouse (2010) (0)
Monitoring the fate of transplanted cells in vivo (1992) (0)
In vivo restoration of dystrophin expression (2005) (0)
Faculty Opinions recommendation of Gene therapy for Duchenne muscular dystrophy: balancing good science, marginal efficacy, high emotions and excessive cost. (2019) (0)
Splice switching as a treatment for Duchenne muscular dystrophy (2009) (0)
Faculty Opinions recommendation of Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy. (2019) (0)
A Review of the Type-1 Fibrillinopathies: Pathophysiology, Diagnosis and Novel Therapeutic Strategies (2018) (0)
O10 Development of PMO antisense oligonucleotides for treatment of Spinal muscular atrophy (2014) (0)
Induced splice-switching to study dystrophin isoform function and expression (2010) (0)
P.6.5 Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing (2013) (0)
Systematic dystrophin expression in the MDX mouse: Morphs make more sense (2006) (0)
Alternate Pax7 alleles are associated with differences in the ability to regenerate damaged skeletal muscle (1998) (0)
T.P.1.08 The use of antisense oligomer for splice switching in spinal muscular atrophy fibroblasts (2009) (0)
Antisense molecules and methods for the treatment of pathologies (2010) (0)
Exceptions to exceptions in the Duchenne: Becker dystrophin reading frame (2013) (0)
P.11.5 PMO-mediated dystrophin exon 23 skipping restores mitochondrial function in the mdx mouse heart (2013) (0)
Morpholino Oligomer Peptide Therapy Improves Mitochondrial Function in mdx Cardiomyopathy (2015) (0)
Corrigendum: Analysis of Pathogenic Pseudoexons Reveals Novel Mechanisms Driving Cryptic Splicing (2022) (0)
Overview of molecular strategies (2007) (0)
Stem cells - a 20th Century Panacea? (2003) (0)
G.P.78 Dystrophin isoforms with incomplete β dystroglycan and syntrophin binding domains retain partial function (2012) (0)
Antisense mediated exon selection to treat human disease (2014) (0)
Antisense oligonucleotides and Exon Skipping: Does size matter? (2005) (0)
Multiple exon skipping in the dystrophin gene: A more effective therapy? (2003) (0)
Faculty Opinions recommendation of Nucleic acid binding proteins affect the subcellular distribution of phosphorothioate antisense oligonucleotides. (2019) (0)
T.P.30 Characterisation of two DMD-causing splice-site mutations and development of personalised exon skipping strategies (2012) (0)
Faculty Opinions recommendation of Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease. (2019) (0)
P26 Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping (2010) (0)
P5.36 Standardized exercise test for identification of children with mitochondrial disorders (2011) (0)
Genetic therapies for SMA when, which treatment and what benefit (2011) (0)
Redirecting dystrophin pre-mRNA splicing using antisense oligonucleotides: An overview (2003) (0)
P18 Transient mouse models for the preclinical evaluation of therapeutic dystrophin exon skipping strategies (2011) (0)
Antisense mediated exon selection to alter gene expression in the study of Disease (2014) (0)
A personalised genetic treatment for DMD (2007) (0)
Faculty Opinions recommendation of Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. (2019) (0)
Faculty Opinions recommendation of Antisense Oligonucleotide-Based Splice Correction of a Deep-Intronic Mutation in CHM Underlying Choroideremia. (2019) (0)
T.P.2.01 Antisense oligomer design: Targeting and assay systems (2008) (0)
Antisense oligomer therapies for neuromuscular disorders (2012) (0)
Golden retriever muscular dystrophy and the development of genetherapy regimes for duchenne muscular dystrophy (1998) (0)
Exon skipping strategies to address DMD-causing mutations: Personalized genetic therapies (2009) (0)
Redirecting pre-mrna splicing using antisense oligonucleotides (2003) (0)
Restoration of dystrophin expression in the mdx mouse using antisense oligonucleotides in a gene "knock-in" approach (2001) (0)
Antisense exon skipping workshop (2005) (0)
Optimisation of antisense oligonucleotide cocktails using in silico and in vitro techniques for targeted exon skipping in the dystrophin central rod domain (2009) (0)
In vitro modification of Tissue-Specific alternative splicing of the mouse TPM3 gene with antisense oligonucleotides (2005) (0)
P4.30 Multiple exon skipping strategies to by-pass selected dystrophin mutations (2011) (0)
Evidence for alternate Pax7 transcripts in skeletal muscle, brain and other organs of adult mice (1997) (0)
Use of the GRMD model to study gene and genetic therapies for muscular dystrophy (1996) (0)
Snap-back SSCP: specific introduction of conformational changes for mutation detection (1995) (0)
Primary Nasal Epithelial Cells As a Type-II Alveolar Surrogate Cell Culture Model for ABCA-3 Deficiency (2021) (0)
Antisense oligonucleotide delivery to cardiac muscle: Knocking on a locked door (2007) (0)
A comprehensive mutation screening strategy of the dystrophin gene of DMD patients preceding antisense oligoribonucleotide clinical trials (2005) (0)
Faculty Opinions recommendation of Severe persistent injection site reactions after subcutaneous 2'-O-methyl phosphorothioate oligonucleotide therapy for Duchenne muscular dystrophy. (2019) (0)
T.P.32 Developing a transient mouse model of Spinal Muscular Atrophy to determine the functionality of SMN on peripheral tissue (2012) (0)
Antisense oligonucleotides for induction of exon omission and methods of use thereof (2005) (0)
Evaluating new therapies & clinical trials: Why, when, which treatment and what benefit? (2010) (0)
Northern analysis of regenerative activity in crush injured and dystrophic (mdx) muscle (1992) (0)
Splice intervention to treat duchenne muscular dystrophy and beyond (2009) (0)
Implementation of splice switching therapies for duchenne muscular dystrophy (2010) (0)
Transient suppression of the Myostatin gene transcript: A comparison of different antisense nucleic acid mechanisms (2003) (0)
Evidence that Pax7 contains two polymorphic homeoboxes, one of which has a rearrangement associated with enhanced skeletal muscle regrowth (1997) (0)
Gene therapy trials in Golden Retriever muscular dystrophy (1999) (0)
T.P.2.09 Induced exon skipping in normal and mdx muscle (2008) (0)
Antisense therapy for inherited neuromuscular disorers (2013) (0)
Characterising splicing defects of ABCA4 variants within exons 13-50 in patient-derived fibroblasts. (2022) (0)
Splice-switching as a treatment for inherited disorders (2011) (0)
P4.31 Transient mouse models for the preclinical evaluation of therapeutic dystrophin exon skipping strategies (2011) (0)
New therapeutic modalities: Opportunities and challenges (2008) (0)
G.P.64 Histological, immunohistochemical and electron microscopic study of muscle in MCK-βAPP mice (2012) (0)
Theme Four: Genetics (2009) (0)
Faculty Opinions recommendation of Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle. (2019) (0)
T.P.42 Antisense oligonucleotide induced over-expression of progerin in human myoblasts: A possible model of muscle aging (2012) (0)
Splice switching therapies as personalised genetic treatments: Applications to muscular dystrophy, thalassemia and spinal muscular atrophy (2009) (0)
Cryptic U2-dependent Pre-mRNASplice Site Usage Induced by Splice Switching Antisense Oligonucleotides (2021) (0)
Altered myogenesis and premature senescence underlie human TRIM32-related myopathy (2019) (0)
Redirecting splicing to restore dystrophin expression in a canine model of muscular dystrophy (2005) (0)
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 (2011) (0)
Faculty Opinions recommendation of Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. (2019) (0)
Faculty Opinions recommendation of Chronic Toxicity Assessment of 2'-O-Methoxyethyl Antisense Oligonucleotides in Mice. (2019) (0)
Regulation of eukaryotic gene expression by the untranslated gene regions and other non-coding elements (2012) (0)
DNAzymes: Expanding the Potential of Nucleic Acid Therapeutics. (2023) (0)
Splice modulating antisense oligonucleotides restore some acid-alpha-glucosidase activity in cells derived from patients with late-onset Pompe disease (2020) (0)
Use of the Golden Retriever dog model for Duchenne muscular dystrophy in gene therapy trials (1997) (0)
Antisense oligonucleotides for induction of exon skipping-and procedures for use thereof (2005) (0)
A molecular therapy for muscular dystrophy (2006) (0)
Comparative expression of myogenic transcription factors in regenerating skeletal muscles from BALB/c and SJL/J mice (1994) (0)
Embryonic stem cells and the fountain of youth: Fact or fantasy (2002) (0)
Antisense induced exon skipping restores dystrophin in the 4CV mouse model of muscular dystrophy (2007) (0)
T.P.28 Pseudo-exon inactivation of the dystrophin gene: Ideal candidates for exon skipping (2012) (0)
Exon skipping and Duchenne muscular dystrophy: A clinical trial update (2013) (0)
Inducing dystrophin pre-mRNA exon skipping as a potential genetic therapy: target selection for improved antisense oligonucleotide activity (2003) (0)
Evidence for multiple functions of PAX 7 (1998) (0)
Adenovirus-mediated myophosphorylase gene transfer in myophosphorylase-deficient cells in vitro (1998) (0)
Preferred antisense oligonucleotide chemistries to overcome dystrophin mutations (2004) (0)
Therapeutic alternative splicing: an update on Duchenne muscular dystrophy clinical trials and other applications (2015) (0)
Rescuing CFTR function caused by mutation specific polymorphisms in cystic fibrosis patients (2018) (0)
Faculty Opinions recommendation of Intracerebroventricular Administration of a 2'-O-Methyl Phosphorothioate Antisense Oligonucleotide Results in Activation of the Innate Immune System in Mouse Brain. (2019) (0)
Faculty Opinions recommendation of Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice. (2019) (0)
986 EVALUATING THE PATIENT-PHYSICIAN RELATIONSHIP IN CHRONIC HEPATITIS B: RESULTS FROM A QUALITATIVE EUROPEAN PATIENT SURVEY (2012) (0)
Faculty Opinions recommendation of Targeting RNA: A transformative therapeutic strategy. (2019) (0)
P.19.2 Targeting the Huntingtin gene with splice-switching AOs: An unusual result (2013) (0)
Making sense in Duchenne muscular dystrophy: Slowing the natural progression of muscle wasting (2017) (0)
Exploring novel therapeutic chemistries in exon-skipping for duchenne muscular dystrophy (2018) (0)
P.6.6 Splice switching Antisense Oligonucleotides: A “kink” in the design? (2013) (0)
Normalisation of gene expression in MDX mice muscle (2009) (0)
Exon skipping therapies to address DMD-causing mutations targeting exon blocks (2009) (0)
Expression of MyoD and myogenin genes in regenerating and dystrophic muscles of mice (1994) (0)
Personalised genetic medicines: Hope and Hype (2014) (0)
Molecular therapies for muscular dystrophy: An overview (2005) (0)
Correction: Corrigendum: Response to “Railroading at the FDA” (2017) (0)
Therapeutic alternative splicing: Opportunities and challenges (2014) (0)
Treatment of Adult mdx Mice with Phosphorodiamidate Morpholino Oligomer Restores Cardiac Mitochondrial Energetics and Prevents the Dystrophic Cardiomyopathy (2017) (0)
Induced non-productive splicing to study gene expression (2009) (0)
Optimising morpholino oligomer therapy in the treatment of mdx cardiomyopathy (2015) (0)
Splicing manipulation for the treatment of duchenne muscular dystrophy (2007) (0)
Late onset Pompe disease: Rescue of acid alpha-glucosidase expression by splice modification (2018) (0)
Genetic probes for tracking muscle precursor cells in vivo: technical aspects (1992) (0)
Faculty Opinions recommendation of RNA Reduction and Hepatotoxic Potential Caused by Non-Gapmer Antisense Oligonucleotides. (2019) (0)
Gene therapy trials in the GRMD model (1998) (0)
Splice-switching as a treament for duchenne muscular dystrophy (2010) (0)
Molecular therapies for the treatment of muscular dystrophy (2004) (0)
Gene therapy trials in the ovine model of McArdle's disease (2001) (0)
Antisense oligomer induced splice manipulation of Survival Motor Neuron exon 7 (2009) (0)
Antisense oligonnucleotide treatment of COL7A1 causes non-specific splice modifications (2018) (0)
Targeted therapeutic alternative splicing (2014) (0)
Personalised genetic medicines for inherited disorders (2014) (0)
Investigations of gene therapy for McArdle's disease using the sheep model (1999) (0)
Splice-modifying antisense oligomers: Precision medicines to correct aberrant splicing (2015) (0)
ANTISENSE OLIGONUCLEOTIDES TO IMPROVE CFTR FUNCTION FOR PEOPLE WITH THE INTRON 9 5T POLYMORPHISM (2019) (0)
Manipulating gene expression using antisense oligonucleotides: A strategy to bypass disease causing mutations (2004) (0)
Endosomolytically active lipids for non-viral gene therapy. (2004) (0)
T.P.33 A cutting-edge approach to Spinal Muscular Atrophy treatment using antisense oligonucleotides (2012) (0)
Antisense therapies for DMD and SMA (2012) (0)
A spanner in the works of human Transformer-2-β1 autoregulation may reduce the severity of spinal muscular atrophy (2015) (0)
Treatment of mdx Mice with Morpholino Oligomers Improves Metabolic Function and Contractility (2016) (0)
Quantitation of growth and regenerative activity in skeletal muscle (1991) (0)
Characterisation of a surrogate Type-II alveolar cell culture model for ATP binding cassette subfamily a member 3 (ABCA-3) deficiency (2020) (0)
Restoring CFTR using antisense oligonucleotides for a cystic fibrosis causing splice mutation C.2989-1G>A (2020) (0)
Multiple exon skipping to correct duplications in the dystrophin gene (2018) (0)
Faculty Opinions recommendation of Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs. (2019) (0)
Faculty Opinions recommendation of Anchor peptide captures, targets, and loads exosomes of diverse origins for diagnostics and therapy. (2019) (0)
In vivo restoration of dystrophin expression by morpholino antisense oligonucleotides (2005) (0)
Muscle biology genes and disease. Therapies for muscular dystrophy: An overview (2008) (0)
Faculty Opinions recommendation of Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps. (2019) (0)
Remarkable clinical trials on boys suffering from Duchene Muscular Dystrophy (2013) (0)
T.P.1.06 Induced non-productive splicing to study muscle gene expression (2009) (0)
NEAT1 polyA-modulating antisense oligonucleotides reveal opposing functions for both long non-coding RNA isoforms in neuroblastoma (2020) (0)
Antisense Oligonucleotide Induction of the hnRNPA1b Isoform Affects Pre-mRNA Splicing of SMN2 in SMA Type I Fibroblasts (2022) (0)
PCR‐based strategies for delimiting large mutations in the DMD gene (2015) (0)
Faculty Opinions recommendation of Injection site reactions after subcutaneous oligonucleotide therapy. (2019) (0)
Transcriptome patterns show normalization of muscle gene expression after restoration of dystrophin in MDX mice by exon skipping (2009) (0)
T.P.2.03 Characterisation of a complex dystrophin mutation: Assume nothing when designing exon skipping strategies (2008) (0)
Induced exon skipping: AO design is more important than chemistry (2007) (0)
Faculty Opinions recommendation of Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease. (2019) (0)
Breakpoint junction features of seven DMD deletion mutations (2019) (0)
Primary Nasal Epithelial Cells as a Surrogate Cell Culture Model for Type-II Alveolar Cells to Study ABCA-3 Deficiency (2022) (0)
Small chemical modulation of induced alternative splicing (2018) (0)

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