Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice. - Wikidata - wikimedia - TriplyDB

Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.

Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.

http://www.wikidata.org/entity/Q34556906

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Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives 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 Splicing therapy for neuromuscular disease Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.Dystrophic Cardiomyopathy-Potential Role of Calcium in Pathogenesis, Treatment and Novel Therapies Evaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx mice Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.Dystromirs as serum biomarkers for monitoring the disease severity in Duchenne muscular Dystrophy.Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy.Could exon skipping help dystrophic boys to run, hop, and jump?Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse Musculoskeletal Geometry, Muscle Architecture and Functional Specialisations of the Mouse Hindlimb Cationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice.Membrane-stabilizing copolymers confer marked protection to dystrophic skeletal muscle in vivo.Elusive sources of variability of dystrophin rescue by exon skipping Behavioral and locomotor measurements using an open field activity monitoring system for skeletal muscle diseases.Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.Circulating Biomarkers for Duchenne Muscular Dystrophy.Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells.Opportunities and challenges for the development of antisense treatment in neuromuscular disorders.Targeting RNA to treat neuromuscular disease.Cell-matrix interactions in muscle disease.Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Drug Discovery of Therapies for Duchenne Muscular Dystrophy.Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy.Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models.Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne.Myoblots: dystrophin quantification by in-cell western assay for a streamlined development of Duchenne muscular dystrophy (DMD) treatments.Ryanodine channel complex stabilizer compound S48168/ARM210 as a disease modifier in dystrophin-deficient mdx mice: proof-of-concept study and independent validation of efficacy.Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice.

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P2860

Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.

http://www.wikidata.org/entity/Q34556906

description

2010 nî lūn-bûn

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2010 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած

@hyw

2010 թվականի նոյեմբերին հրատարակված գիտական հոդված

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2010年の論文

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2010年論文

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2010年論文

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2010年論文

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2010年論文

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2010年論文

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2010年论文

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Chronic systemic therapy with ...... ocomotor behavior in mdx mice.

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Chronic systemic therapy with ...... ocomotor behavior in mdx mice.

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Chronic systemic therapy with ...... ocomotor behavior in mdx mice.

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Chronic systemic therapy with ...... ocomotor behavior in mdx mice.

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Chronic systemic therapy with ...... ocomotor behavior in mdx mice.

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Molecular Therapy

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Chronic systemic therapy with ...... ocomotor behavior in mdx mice.

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Alberto Malerba

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Francesco Muntoni

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George Dickson

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Ian R Graham

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Paul S Sharp

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P2860

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

X chromosome-linked muscular dystrophy (mdx) in the mouse

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.

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

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

Preclinical drug trials in the mdx mouse: assessment of reliable and sensitive outcome measures.

Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide.

Vivo-Morpholinos: a non-peptide transporter delivers Morpholinos into a wide array of mouse tissues.

Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy.

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

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10.1038/MT.2010.261

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2

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Virginia Arechavala-Gomeza

Dominic J Wells

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49631106

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