| scholarly article | Q13442814 |
| P50 | author | Gert-Jan van Ommen | Q29643727 |
| Frank Baas | Q30111686 | ||
| Judith van Deutekom | Q130284748 | ||
| P2093 | author name string | Johan T den Dunnen | |
| Annemieke Aartsma-Rus | |||
| Wendy E Kaman | |||
| Anneke A M Janson | |||
| Mattie Bremmer-Bout | |||
| P433 | issue | 8 | |
| P921 | main subject | patient | Q181600 |
| P304 | page(s) | 907-914 | |
| P577 | publication date | 2003-04-01 | |
| P1433 | published in | Human Molecular Genetics | Q2720965 |
| P1476 | title | Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. | |
| P478 | volume | 12 |
| Q41877911 | A novel dysferlin mutant pseudoexon bypassed with antisense oligonucleotides |
| Q50018682 | A novel human muscle cell model of Duchenne muscular dystrophy created by CRISPR/Cas9 and evaluation of antisense-mediated exon skipping |
| Q34267400 | Advances in Duchenne muscular dystrophy gene therapy |
| Q45874519 | Ameliorating pathogenesis by removing an exon containing a missense mutation: a potential exon-skipping therapy for laminopathies. |
| Q55284165 | Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts. |
| Q28475196 | Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient |
| Q42122236 | Antisense mediated exon skipping therapy for duchenne muscular dystrophy (DMD) |
| Q27480935 | Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries |
| Q45858623 | Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. |
| Q55018458 | Antisense oligonucleotides in neurological disorders. |
| Q39880027 | Antisense therapeutics for neurofibromatosis type 1 caused by deep intronic mutations |
| Q34608717 | Antisense therapy in neurology |
| Q33289993 | Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy |
| Q41944520 | Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense |
| Q24682555 | Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications |
| Q34538171 | Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain |
| Q33388537 | Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy |
| Q39645240 | BMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse model |
| Q59876279 | Biomarkers of Duchenne muscular dystrophy: current findings |
| Q37807346 | CPP-Directed Oligonucleotide Exon Skipping in Animal Models of Duchenne Muscular Dystrophy |
| Q46375612 | Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. |
| Q45874288 | Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database. |
| Q50999350 | Clinical characterisation of Becker muscular dystrophy patients predicts favourable outcome in exon-skipping therapy. |
| Q44960581 | Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells |
| Q35694248 | Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides. |
| Q34994545 | Current status of pharmaceutical and genetic therapeutic approaches to treat DMD. |
| Q47440527 | Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy |
| Q36632942 | De novo mutations in neurological and psychiatric disorders: effects, diagnosis and prevention |
| Q33625875 | Delivery is key: lessons learnt from developing splice-switching antisense therapies |
| Q38357434 | Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene. |
| Q36128670 | Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy. |
| Q33877924 | Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy |
| Q35911460 | Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment |
| Q34278933 | Dystrophin and mutations: one gene, several proteins, multiple phenotypes |
| Q33930110 | Dystrophin isoform induction in vivo by antisense-mediated alternative splicing. |
| Q34711962 | Effective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient mice |
| Q41520323 | Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model |
| Q58568817 | Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy |
| Q39392603 | Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy |
| Q24631244 | Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy |
| Q28831088 | Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy |
| Q47115483 | Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne. |
| Q56990656 | Exon Skipping in a Dysf-Missense Mutant Mouse Model |
| Q39692533 | Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping |
| Q42962196 | Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations |
| Q37035750 | Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy. |
| Q38014906 | Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients? |
| Q57639981 | Exon skipping-mediated dystrophin reading frame restoration for small mutations |
| Q83925361 | Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons |
| Q34111572 | Female human iPSCs retain an inactive X chromosome |
| Q45861345 | Functional Characterization and Rescue of a Deep Intronic Mutation in OCRL Gene Responsible for Lowe Syndrome. |
| Q42412768 | Functional rescue of dystrophin-deficient mdx mice by a chimeric peptide-PMO. |
| Q54609129 | Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy. |
| Q89452029 | Gene therapies for axonal neuropathies: Available strategies, successes to date, and what to target next |
| Q35184730 | Gene therapy for muscular dystrophy - a review of promising progress |
| Q24680275 | Gene therapy in clinical medicine |
| Q37396408 | Gene therapy in large animal models of muscular dystrophy |
| Q34576814 | Gene therapy progress and prospects: Duchenne muscular dystrophy |
| Q58223916 | Generation and Characterization of Transgenic Mice with the Full-length HumanDMDGene |
| Q34405099 | Genetic analysis of three genes causing isolated methylmalonic acidemia: identification of 21 novel allelic variants |
| Q33411557 | Genetic correction of splice site mutation in purified and enriched myoblasts isolated from mdx5cv mice |
| Q64227382 | Genotypes and Phenotypes of Small Mutations in Chinese Patients With Dystrophinopathies |
| Q34792390 | High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics |
| Q64069475 | Human induced pluripotent stem cell models for the study and treatment of Duchenne and Becker muscular dystrophies |
| Q47158837 | Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy. |
| Q36972091 | Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. |
| Q64934843 | In Vitro Multiexon Skipping by Antisense PMOs in Dystrophic Dog and Exon 7-Deleted DMD Patient. |
| Q34343780 | In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse |
| Q82604893 | Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers |
| Q27438151 | Inhibitory control over Ca(2+) sparks via mechanosensitive channels is disrupted in dystrophin deficient muscle but restored by mini-dystrophin expression |
| Q35014916 | Interventions for muscular dystrophy: molecular medicines entering the clinic |
| Q83181725 | Intravenous infusion of an antisense oligonucleotide results in exon skipping in muscle dystrophin mRNA of Duchenne muscular dystrophy |
| Q28598287 | Investigating Synthetic Oligonucleotide Targeting of Mir31 in Duchenne Muscular Dystrophy |
| Q84480135 | Less is more: therapeutic exon skipping for Duchenne muscular dystrophy |
| Q24643018 | Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study |
| Q33435110 | Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study |
| Q35802092 | Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping |
| Q36179776 | MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy. |
| Q47438944 | Making sense of antisense oligonucleotides: A narrative review |
| Q37120427 | Mice with missense and nonsense NF1 mutations display divergent phenotypes compared with human neurofibromatosis type I. |
| Q36866549 | Modulating the expression of disease genes with RNA-based therapy. |
| Q36907560 | Molecular therapeutic strategies targeting Duchenne muscular dystrophy |
| Q37142004 | Molecular-targeted therapy for Duchenne muscular dystrophy: progress and potential |
| Q80501077 | Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse |
| Q36116293 | Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort |
| Q47584439 | Myoblots: dystrophin quantification by in-cell western assay for a streamlined development of Duchenne muscular dystrophy (DMD) treatments |
| Q33326762 | Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice |
| Q37814437 | New insights in gene-derived therapy: the example of Duchenne muscular dystrophy |
| Q64104371 | Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results |
| Q40053602 | Oligonucleotidic therapeutics |
| Q37867701 | Opportunities and challenges for the development of antisense treatment in neuromuscular disorders |
| Q96126745 | Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy |
| Q39882118 | Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse. |
| Q92252589 | Phenotype predictions for exon deletions/duplications: A user guide for professionals and clinicians using Becker and Duchenne muscular dystrophy as examples |
| Q35084227 | Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice. |
| Q37338790 | Polymersome delivery of siRNA and antisense oligonucleotides. |
| Q39880997 | Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model |
| Q42587467 | Prednisolone treatment does not interfere with 2'-O-methyl phosphorothioate antisense-mediated exon skipping in Duchenne muscular dystrophy |
| Q37767638 | Present and future of antisense therapy for splicing modulation in inherited metabolic disease |
| Q34734708 | Progress in muscular dystrophy research with special emphasis on gene therapy |
| Q37609948 | Progress in therapeutic antisense applications for neuromuscular disorders |
| Q36512118 | Propionic and methylmalonic acidemia: antisense therapeutics for intronic variations causing aberrantly spliced messenger RNA |
| Q49196789 | RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect. |
| Q90431791 | Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies |
| Q26776412 | Regulation of mammalian transcription and splicing by Nuclear RNAi |
| Q34209715 | Rescue of a dystrophin-like protein by exon skipping in vivo restores GABAA-receptor clustering in the hippocampus of the mdx mouse. |
| Q45033314 | Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study |
| Q41106058 | Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes |
| Q38089634 | Silencing human genetic diseases with oligonucleotide-based therapies. |
| Q45007187 | Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides |
| Q37695543 | Targeted exon skipping to correct exon duplications in the dystrophin gene. |
| Q28478166 | Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development |
| Q39154733 | Targeting Splicing in the Treatment of Human Disease |
| Q37082470 | Technology insight: therapy for Duchenne muscular dystrophy-an opportunity for personalized medicine? |
| Q35508913 | The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations |
| Q28484390 | The effect of 6-thioguanine on alternative splicing and antisense-mediated exon skipping treatment for duchenne muscular dystrophy |
| Q26769701 | The importance of genetic diagnosis for Duchenne muscular dystrophy |
| Q36963079 | The potential of exon skipping for treatment for Duchenne muscular dystrophy |
| Q34926082 | Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations |
| Q49961986 | Therapeutic Targeting of Long Non-Coding RNAs in Cancer |
| Q34329418 | Therapeutic exon skipping for dysferlinopathies? |
| Q38306997 | Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides |
| Q37363171 | Therapeutic potential of splice-switching oligonucleotides |
| Q36549699 | Therapeutic restoration of dystrophin expression in Duchenne muscular dystrophy. |
| Q35952733 | Therapeutic strategies for Duchenne and Becker dystrophies |
| Q38996657 | Translational development of splice-modifying antisense oligomers |
| Q24608004 | miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy |
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