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Antisense-mediated exon skipping: a versatile tool with therapeutic and research applicationsThe importance of genetic diagnosis for Duchenne muscular dystrophyThe effect of 6-thioguanine on alternative splicing and antisense-mediated exon skipping treatment for duchenne muscular dystrophyGuidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translationalTargeting several CAG expansion diseases by a single antisense oligonucleotideEvaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular DystrophyLow dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout miceAntisense-induced exon skipping for duplications in Duchenne muscular dystrophyAssessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophyTargeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor.Delivery is key: lessons learnt from developing splice-switching antisense therapiesMimicking Cardiac Fibrosis in a Dish: Fibroblast Density Rather than Collagen Density Weakens Cardiomyocyte FunctionPrevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review.Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy.Affinity proteomics within rare diseases: a BIO-NMD study for blood biomarkers of muscular dystrophiesPhage display screening without repetitious selection rounds.Assessing functional performance in the mdx mouse modelThe effects of low levels of dystrophin on mouse muscle function and pathologySystemic administration of PRO051 in Duchenne's muscular dystrophy.Advances in therapeutic RNA-targeting.Therapeutic exon skipping for dysferlinopathies?Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule.Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain.Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressivaTheoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.Generation of embryonic stem cells and mice for duchenne research.Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.Autophagy is Impaired in the Tibialis Anterior of Dystrophin Null MicePeptide conjugation of 2'-O-methyl phosphorothioate antisense oligonucleotides enhances cardiac uptake and exon skipping in mdx mice.The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations.Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trialsPreclinical studies on intestinal administration of antisense oligonucleotides as a model for oral delivery for treatment of duchenne muscular dystrophy.Imperatives for DUCHENNE MD: a Simplified Guide to Comprehensive Care for Duchenne Muscular DystrophyAntisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy.SplicePie: a novel analytical approach for the detection of alternative, non-sequential and recursive splicing.Non-sequential and multi-step splicing of the dystrophin transcript.Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants.The NOTCH3 score: a pre-clinical CADASIL biomarker in a novel human genomic NOTCH3 transgenic mouse model with early progressive vascular NOTCH3 accumulationInhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP).The dystrophin gene and cognitive function in the general population.
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Annemieke Aartsma-Rus
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