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

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

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http://www.wikidata.org/entity/Q34035676

Q34035676

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miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy Telomerase RNA level limits telomere maintenance in X-linked dyskeratosis congenita Doxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettes Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping Gene-mediated restoration of normal myofiber elasticity in dystrophic muscles.Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.Repair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy.Therapeutic approaches to muscular dystrophy.The interplay between microRNAs and the neurotrophin receptor tropomyosin-related kinase C controls proliferation of human neuroblastoma cells.MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy.Emerging strategies for cell and gene therapy of the muscular dystrophies Splice-switching antisense oligonucleotides as therapeutic drugs Gene therapy for muscular dystrophy: current progress and future prospects.Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Genome mapping and expression analyses of human intronic noncoding RNAs reveal tissue-specific patterns and enrichment in genes related to regulation of transcription.Longitudinal MRI quantification of muscle degeneration in Duchenne muscular dystrophy.Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery.Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping.Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts.Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations.Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense.Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells.Impact, Characterization, and Rescue of Pre-mRNA Splicing Mutations in Lysosomal Storage Disorders.

P2860

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P2860

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

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http://www.wikidata.org/entity/Q34035676

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2002年の論文

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