Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through. - Wikidata - awgldk - TriplyDB

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.

http://www.wikidata.org/entity/Q35072755

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Experimental therapies and ongoing clinical trials to slow down progression of ADPKD Targeting mRNA for the treatment of facioscapulohumeral muscular dystrophy Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.Global and disease-associated genetic variation in the human Fanconi anemia gene family.Antisense therapy in neurology VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects.Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy The use of urinary and kidney SILAM proteomics to monitor kidney response to high dose morpholino oligonucleotides in the mdx mouse.Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.Elusive sources of variability of dystrophin rescue by exon skipping CD49d is a disease progression biomarker and a potential target for immunotherapy in Duchenne muscular dystrophy Engraftment of ES-Derived Myogenic Progenitors in a Severe Mouse Model of Muscular Dystrophy.Accurate Quantitation of Dystrophin Protein in Human Skeletal Muscle Using Mass Spectrometry Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice.Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy.Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy Current Translational Research and Murine Models For Duchenne Muscular Dystrophy.Generation of skeletal muscle cells from embryonic and induced pluripotent stem cells as an in vitro model and for therapy of muscular dystrophies.Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?Duchenne muscular dystrophy drug discovery - the application of utrophin promoter activation screening.Cardiac and respiratory dysfunction in Duchenne muscular dystrophy and the role of second messengers.New developments in exon skipping and splice modulation therapies for neuromuscular diseases.Drug Discovery of Therapies for Duchenne Muscular Dystrophy.Pathoproteomic profiling of the skeletal muscle matrisome in dystrophinopathy associated myofibrosis.Evolving paradigms in clinical pharmacology and therapeutics for the treatment of Duchenne muscular dystrophy.Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice.Eteplirsen for the treatment of Duchenne muscular dystrophy.Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping.Flow Cytometry-Defined CD49d Expression in Circulating T-Lymphocytes Is a Biomarker for Disease Progression in Duchenne Muscular Dystrophy.Molecular diagnosis of dystrophinopathies using a multi-technique analysis algorithm.Skipping Multiple Exons to Treat DMD-Promises and Challenges.Assisted delivery of antisense therapeutics in animal models of heritable neurodegenerative and neuromuscular disorders: a systematic review and meta-analysis.Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD

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P2860

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.

http://www.wikidata.org/entity/Q35072755

description

2011 nî lūn-bûn

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2011 թուականի Մայիսին հրատարակուած գիտական յօդուած

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2011 թվականի մայիսին հրատարակված գիտական հոդված

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2011年の論文

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2011年論文

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2011年論文

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2011年論文

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Restoring dystrophin expressio ...... g and stop codon read through.

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Restoring dystrophin expressio ...... g and stop codon read through.

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Restoring dystrophin expressio ...... g and stop codon read through.

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Restoring dystrophin expressio ...... g and stop codon read through.

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Restoring dystrophin expressio ...... g and stop codon read through.

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Restoring dystrophin expressio ...... g and stop codon read through.

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J.AJPATH.2011.03.050

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The American Journal of Pathology

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Restoring dystrophin expressio ...... g and stop codon read through.

@en

P2093

Abby Bronson

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Andreas R Baudy

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Arthur A Levin

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Edward M Connor

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Eric P Hoffman

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Shin'ichi Takeda

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Toshifumi Yokota

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P2860

Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy

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

Morpholino antisense oligomers: design, preparation, and properties

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

Duchenne dystrophy: electron microscopic findings pointing to a basic or early abnormality in the plasma membrane of the muscle fiber.

A phase I trial of aprinocarsen (ISIS 3521/LY900003), an antisense inhibitor of protein kinase C-alpha administered as a 24-hour weekly infusion schedule in patients with advanced cancer.

Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo.

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12-22

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10.1016/J.AJPATH.2011.03.050

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1

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179

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2011-05-23T00:00:00Z

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251446539

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21703390

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3124804

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