| scholarly article | Q13442814 |
| P2093 | author name string | Manoharan M | |
| Dickson G | |||
| Dunckley MG | |||
| Eperon IC | |||
| Villiet P | |||
| P433 | issue | 7 | |
| P304 | page(s) | 1083-1090 | |
| P577 | publication date | 1998-07-01 | |
| P1433 | published in | Human Molecular Genetics | Q2720965 |
| P1476 | title | Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides | |
| P478 | volume | 7 |
| Q33916133 | 2'-carbohydrate modifications in antisense oligonucleotide therapy: importance of conformation, configuration and conjugation |
| Q52688621 | A dystrophic Duchenne mouse model for testing human antisense oligonucleotides. |
| Q39350652 | A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice |
| Q33950888 | A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground |
| Q38303896 | A targeted oligonucleotide enhancer of SMN2 exon 7 splicing forms competing quadruplex and protein complexes in functional conditions |
| Q45730194 | Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product |
| Q34267400 | Advances in Duchenne muscular dystrophy gene therapy |
| Q38542416 | Advances in genetic therapeutic strategies for Duchenne muscular dystrophy |
| Q44879918 | Alternative splicing in disease and therapy |
| Q28084979 | Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy |
| Q38734734 | Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease. |
| Q47344432 | Antisense oligonucleotides: the next frontier for treatment of neurological disorders |
| Q41944520 | Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense |
| Q38340328 | Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment. |
| Q24682555 | Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications |
| Q34574529 | Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. |
| Q37629659 | Cell penetrating peptides: overview and applications to the delivery of oligonucleotides |
| Q37631779 | Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells. |
| Q37907407 | Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy. |
| Q34035676 | Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells |
| Q92434208 | Commentary: SU9516 increases α7β1 Integrin and Ameliorates Disease Progression in the mdx Mouse Model of Duchenne Muscular Dystrophy |
| Q44960581 | Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells |
| Q58830804 | Conjugation of PEG and gold nanoparticles to increase the accessibility and valency of tethered RNA splicing enhancers |
| Q24679378 | Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy |
| Q42807733 | Correction of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides |
| Q38296901 | Correction of alternative splicing of tau in frontotemporal dementia and parkinsonism linked to chromosome 17. |
| Q38318922 | Design principles for bifunctional targeted oligonucleotide enhancers of splicing |
| Q37105210 | Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy |
| Q38087705 | Duchenne muscular dystrophy drug discovery - the application of utrophin promoter activation screening |
| Q44264343 | Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice |
| Q36897347 | Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer |
| Q46087408 | Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs |
| Q30383884 | Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method. |
| Q55496176 | Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers. |
| Q36960845 | Emerging strategies for cell and gene therapy of the muscular dystrophies |
| Q24631244 | Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy |
| Q38014906 | Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients? |
| Q34204198 | Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice |
| Q38186137 | Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases |
| Q36297940 | Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs. |
| Q34180144 | Gene Therapy for Muscular Dystrophies: Progress and Challenges |
| Q33831193 | Gene and cell-mediated therapies for muscular dystrophy. |
| Q54609129 | Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy. |
| Q24680275 | Gene therapy in clinical medicine |
| Q37396408 | Gene therapy in large animal models of muscular dystrophy |
| Q33411557 | Genetic correction of splice site mutation in purified and enriched myoblasts isolated from mdx5cv mice |
| Q33525253 | Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo |
| Q34792390 | High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics |
| Q34973311 | Human Splicing Finder: an online bioinformatics tool to predict splicing signals |
| Q47158430 | Hybrid splicing minigene and antisense oligonucleotides as efficient tools to determine functional protein/RNA interactions. |
| Q33840931 | Immunocytochemical analysis of human muscular dystrophy |
| Q41949953 | Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy. |
| Q36972091 | Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle. |
| 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 |
| Q40919541 | Induction of endogenous Bcl-xS through the control of Bcl-x pre-mRNA splicing by antisense oligonucleotides |
| Q24563176 | Inhibition of HIV-1 multiplication by antisense U7 snRNAs and siRNAs targeting cyclophilin A |
| Q28598287 | Investigating Synthetic Oligonucleotide Targeting of Mir31 in Duchenne Muscular Dystrophy |
| Q33573648 | Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers |
| Q42257279 | Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino |
| 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 |
| Q38296244 | Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3' splice site pairing |
| Q50972527 | Molecular diagnosis of dystrophinopathies using a multi-technique analysis algorithm. |
| Q39342422 | Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy |
| Q37814437 | New insights in gene-derived therapy: the example of Duchenne muscular dystrophy |
| Q34204364 | Novel approaches to treat muscular dystrophies |
| Q36925191 | Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents |
| Q29048207 | Novel long noncoding RNAs (lncRNAs) in myogenesis: a miR-31 overlapping lncRNA transcript controls myoblast differentiation |
| Q35172284 | Novel therapies for Duchenne muscular dystrophy |
| Q37068616 | Nuclear antisense effects in cyclophilin A pre-mRNA splicing by oligonucleotides: a comparison of tricyclo-DNA with LNA |
| Q33713525 | Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice |
| Q34807121 | Oligonucleotide-mediated gene therapy for muscular dystrophies |
| Q34621772 | One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice |
| Q81825063 | Peptide-conjugated antisense therapy takes a skip ahead |
| Q41890992 | Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment. |
| Q30493685 | Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping |
| Q30626615 | Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice |
| Q37949928 | RNA interference for performance enhancement and detection in doping control |
| Q90431791 | Recent advancements in exon-skipping therapies using antisense oligonucleotides and genome editing for the treatment of various muscular dystrophies |
| Q92489474 | Recent advances in Duchenne muscular dystrophy |
| Q38079633 | Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies |
| Q35072755 | Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through |
| Q51065898 | SU9516 Increases α7β1 Integrin and Ameliorates Disease Progression in the mdx Mouse Model of Duchenne Muscular Dystrophy. |
| Q34807136 | Screening for antisense modulation of dystrophin pre-mRNA splicing |
| Q42915987 | Selective inhibition of ADAR2-catalyzed editing of the serotonin 2c receptor pre-mRNA by a helix-threading peptide |
| Q33853846 | Selective modification of alternative splicing by indole derivatives that target serine-arginine-rich protein splicing factors |
| Q38089634 | Silencing human genetic diseases with oligonucleotide-based therapies. |
| Q73741323 | Skipping to new gene therapies for muscular dystrophy |
| Q36088922 | Small regulatory RNAs in mammals |
| Q33868227 | Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides. |
| Q37211004 | Splice-switching antisense oligonucleotides as therapeutic drugs |
| Q36342775 | Splicing regulators: targets and drugs |
| Q44212614 | Systemically delivered antisense oligomers upregulate gene expression in mouse tissues |
| Q52547202 | Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy. |
| Q45007187 | Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides |
| Q28478166 | Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development |
| Q37909000 | Targeting RNA to treat neuromuscular disease |
| Q38301046 | The Rossmann fold of glyceraldehyde-3-phosphate dehydrogenase (GAPDH) is a nuclear docking site for antisense oligonucleotides containing a TAAAT motif. |
| Q34590393 | The influence of antisense oligonucleotide length on dystrophin exon skipping. |
| Q34473336 | The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy |
| Q28818017 | Therapeutic Potential of Tricyclo-DNA antisense oligonucleotides |
| Q37455625 | Therapeutic advances in muscular dystrophy |
| Q34982869 | Therapeutic approaches to muscular dystrophy. |
| Q34329418 | Therapeutic exon skipping for dysferlinopathies? |
| 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 |
| Q35607136 | Therapeutics for Duchenne muscular dystrophy: current approaches and future directions. |
| Q38996657 | Translational development of splice-modifying antisense oligomers |
| Q91841518 | Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice |
| Q38289261 | Viral Vector-Mediated Antisense Therapy for Genetic Diseases |
| Q92532811 | mRNA levels can be reduced by antisense oligonucleotides via no-go decay pathway |
| Q24608004 | miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy |
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