Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches - Wikidata - wikimedia - TriplyDB

Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches

Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches

http://www.wikidata.org/entity/Q42013912

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mRNA trans-splicing in gene therapy for genetic diseases FUBP1: a new protagonist in splicing regulation of the DMD gene Repair of rhodopsin mRNA by spliceosome-mediated RNA trans-splicing: a new approach for autosomal dominant retinitis pigmentosa.The pathogenicity of splicing defects: mechanistic insights into pre-mRNA processing inform novel therapeutic approaches.Reprogramming the Dynamin 2 mRNA by Spliceosome-mediated RNA Trans-splicing.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations.Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3' splice sites.Cis-splicing and translation of the pre-trans-splicing molecule combine with efficiency in spliceosome-mediated RNA trans-splicing Correction of the Middle Eastern M712T mutation causing GNE myopathy by trans-splicing.High-Throughput Assays to Assess the Functional Impact of Genetic Variants: A Road Towards Genomic-Driven Medicine.Targeting Splicing in the Treatment of Human Disease Considerations for a Successful RNA Trans-splicing Repair of Genetic Disorders Normal and altered pre-mRNA processing in the DMD gene.Optimization of trans-Splicing for Huntington's Disease RNA Therapy.Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy.Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.

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Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches

http://www.wikidata.org/entity/Q42013912

description

2013 nî lūn-bûn

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name

Dystrophin rescue by trans-spl ...... e for exon skipping approaches

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Dystrophin rescue by trans-spl ...... e for exon skipping approaches

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Dystrophin rescue by trans-spl ...... e for exon skipping approaches

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Dystrophin rescue by trans-spl ...... e for exon skipping approaches

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Dystrophin rescue by trans-spl ...... e for exon skipping approaches

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Dystrophin rescue by trans-spl ...... e for exon skipping approaches

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Nucleic Acids Research

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Dystrophin rescue by trans-spl ...... e for exon skipping approaches

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Arnaud Jollet

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Graziella Griffith

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Guillaume Précigout

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Kamel Mamchaoui

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Luis Garcia

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Maëva Le Hir

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Susanne Philippi

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Thomas Voit

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Development of a single vector system that enhances trans-splicing of SMN2 transcripts

Heterologous introns can enhance expression of transgenes in mice

Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

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

U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

Interplay between exonic splicing enhancers, mRNA processing, and mRNA surveillance in the dystrophic Mdx mouse

ESEfinder: A web resource to identify exonic splicing enhancers

Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

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10.1093/NAR/GKT621

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17

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41

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Stéphanie Lorain

Cécile Peccate

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2013-07-16T00:00:00Z

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