New developments in exon skipping and splice modulation therapies for neuromuscular diseases.
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Targeting mRNA for the treatment of facioscapulohumeral muscular dystrophySkeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical TrialsGene therapy for the nervous system: challenges and new strategies.In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophyDuchenne muscular dystrophy: current cell therapiesBiomarker for Spinal Muscular Atrophy: Expression of SMN in Peripheral Blood of SMA Patients and Healthy ControlsThe rod domain is not essential for the function of plectin in maintaining tissue integrityNonmechanical Roles of Dystrophin and Associated Proteins in Exercise, Neuromuscular Junctions, and Brains.Exon skipping of FcεRIβ eliminates expression of the high-affinity IgE receptor in mast cells with therapeutic potential for allergy.Current Translational Research and Murine Models For Duchenne Muscular Dystrophy.Developing therapies for spinal muscular atrophy.Longitudinal characterization of biomarkers for spinal muscular atrophy.Genetically engineered livestock for biomedical models.Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.Antisense oligonucleotide drugs for Duchenne muscular dystrophy: how far have we come and what does the future hold?Translational development of splice-modifying antisense oligomers.Human RECQ Helicase Pathogenic Variants, Population Variation and "Missing" Diseases.Alternative Pre-mRNA Splicing of Toll-Like Receptor Signaling Components in Peripheral Blood Mononuclear Cells from ARDS Patients.Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides.Target resequencing of neuromuscular disease-related genes using next-generation sequencing for patients with undiagnosed early-onset neuromuscular disorders.Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.Functional Characterization and Rescue of a Deep Intronic Mutation in OCRL Gene Responsible for Lowe Syndrome.Immortalized Muscle Cell Model to Test the Exon Skipping Efficacy for Duchenne Muscular Dystrophy.Designing Effective Antisense Oligonucleotides for Exon Skipping.Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy.Use of antisense oligonucleotides to correct the splicing error in ISCU myopathy patient cell lines.CUGC for Duchenne muscular dystrophy (DMD).Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy.Skipping Multiple Exons to Treat DMD-Promises and Challenges.Alternative Splicing in the Hippo Pathway-Implications for Disease and Potential Therapeutic Targets.Construction of a tri-chromatic reporter cell line for the rapid and simple screening of splice-switching oligonucleotides targeting DMD exon 51 using high content screening.
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New developments in exon skipping and splice modulation therapies for neuromuscular diseases.
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article científic
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article scientifique
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articol științific
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articolo scientifico
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artigo científico
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artigo científico
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artigo científico
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artikel ilmiah
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artikull shkencor
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artículo científico
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name
New developments in exon skipp ...... es for neuromuscular diseases.
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type
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New developments in exon skipp ...... es for neuromuscular diseases.
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prefLabel
New developments in exon skipp ...... es for neuromuscular diseases.
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P2093
P2860
P1476
New developments in exon skipp ...... es for neuromuscular diseases.
@en
P2093
Aleksander Touznik
Joshua J A Lee
Toshifumi Yokota
P2860
P304
P356
10.1517/14712598.2014.896335
P407
P577
2014-03-12T00:00:00Z