Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice. - Wikidata - wikimedia - TriplyDB

Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice.

Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice.

http://www.wikidata.org/entity/Q33713525

about

Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery A chemical view of oligonucleotides for exon skipping and related drug applications Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy Splicing therapy for neuromuscular disease Rapid screening of gene function by systemic delivery of morpholino oligonucleotides to live mouse embryos Targeting mRNA for the treatment of facioscapulohumeral muscular dystrophy Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy Physico-chemical characteristics of lipoplexes influence cell uptake mechanisms and transfection efficacy Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development The effect of 6-thioguanine on alternative splicing and antisense-mediated exon skipping treatment for duchenne muscular dystrophy Investigating Synthetic Oligonucleotide Targeting of Mir31 in Duchenne Muscular Dystrophy Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo.Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency Evaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx mice Vivo-Morpholino knockdown of alphaIIb: A novel approach to inhibit thrombocyte function in adult zebrafish Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy Emerging genetic therapies to treat Duchenne muscular dystrophy Vascular Endothelial Growth Factor Receptor 1 morpholino increases graft survival in a murine penetrating keratoplasty model.Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver.Physiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy.The status of exon skipping as a therapeutic approach to duchenne muscular dystrophy Targeting protein translation, RNA splicing, and degradation by morpholino-based conjugates in Plasmodium falciparum.Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice.Efficient in vivo manipulation of alternative pre-mRNA splicing events using antisense morpholinos in mice.Vivo-morpholinos induced transient knockdown of physical activity related proteins.Progress in muscular dystrophy research with special emphasis on gene therapy.Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.Duchenne muscular dystrophy gene therapy: Lost in translation?Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice Lessons learned from vivo-morpholinos: How to avoid vivo-morpholino toxicity.Disruption of KATP channel expression in skeletal muscle by targeted oligonucleotide delivery promotes activity-linked thermogenesis Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy.Vascular endothelial growth factor receptor 1 morpholino decreases angiogenesis in a murine corneal suture model.Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice.Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy.

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P2860

Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice.

http://www.wikidata.org/entity/Q33713525

description

2009 nî lūn-bûn

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2009 թուականի Մարտին հրատարակուած գիտական յօդուած

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2009 թվականի մարտին հրատարակված գիտական հոդված

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2009年の論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年论文

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name

Octa-guanidine morpholino rest ...... hology in dystrophic mdx mice.

@ast

Octa-guanidine morpholino rest ...... hology in dystrophic mdx mice.

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type

label

Octa-guanidine morpholino rest ...... hology in dystrophic mdx mice.

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Octa-guanidine morpholino rest ...... hology in dystrophic mdx mice.

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prefLabel

Octa-guanidine morpholino rest ...... hology in dystrophic mdx mice.

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Octa-guanidine morpholino rest ...... hology in dystrophic mdx mice.

@en

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P1433

Molecular Therapy

P1476

Octa-guanidine morpholino rest ...... hology in dystrophic mdx mice.

@en

P2093

Bo Wu

http://www.w3.org/2001/XMLSchema#string

Paul A Morcos

http://www.w3.org/2001/XMLSchema#string

Peijuan Lu

http://www.w3.org/2001/XMLSchema#string

Qi Long Lu

http://www.w3.org/2001/XMLSchema#string

Timothy J Doran

http://www.w3.org/2001/XMLSchema#string

Yongfu Li

http://www.w3.org/2001/XMLSchema#string

P2860

Morpholino antisense oligomers: design, preparation, and properties

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents.

Design and synthesis of dendritic molecular transporter that achieves efficient in vivo delivery of morpholino antisense oligo.

Very mild muscular dystrophy associated with the deletion of 46% of dystrophin.

Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides.

Current treatment of adult Duchenne muscular dystrophy.

Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse.

Transvascular delivery of small interfering RNA to the central nervous system.

Screening for antisense modulation of dystrophin pre-mRNA splicing.

P304

864-871

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scholarly article

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10.1038/MT.2009.38

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5

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17

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2009-03-10T00:00:00Z

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24192124

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19277018

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2835139

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