Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy - Wikidata - wikimedia - TriplyDB

Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy

Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy

http://www.wikidata.org/entity/Q35750055

about

Dystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophies Composite biomarkers for assessing Duchenne muscular dystrophy: an initial assessment.Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.Pharmacologic management of Duchenne muscular dystrophy: target identification and preclinical trials Correlation of Utrophin Levels with the Dystrophin Protein Complex and Muscle Fibre Regeneration in Duchenne and Becker Muscular Dystrophy Muscle Biopsies.Dystrophin quantification: Biological and translational research implications.Antisense therapy in neurology High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics Skeletal muscle fibrosis in the mdx/utrn+/- mouse validates its suitability as a murine model of Duchenne muscular dystrophy.Dystrophin hydrophobic regions in the pathogenesis of Duchenne and Becker muscular dystrophies.Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy.The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials.Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype.Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy.Allele-specific RNA interference rescues the long-QT syndrome phenotype in human-induced pluripotency stem cell cardiomyocytes.Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?Nitric oxide in myogenesis and therapeutic muscle repair.Duchenne muscular dystrophy drug discovery - the application of utrophin promoter activation screening.Oligonucleotide conjugates for therapeutic applications.New Modalities for Challenging Targets in Drug Discovery.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Redirecting splicing with bifunctional oligonucleotides.In vitro correction of a pseudoexon-generating deep intronic mutation in LGMD2A by antisense oligonucleotides and modified small nuclear RNAs.Dystrophin induced cognitive impairment: mechanisms, models and therapeutic strategies Evolving paradigms in clinical pharmacology and therapeutics for the treatment of Duchenne muscular dystrophy.Co-delivery of indoleamine 2,3-dioxygenase prevents loss of expression of an antigenic transgene in dystrophic mouse muscles.Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts.N-terminal α Dystroglycan (αDG-N): A Potential Serum Biomarker for Duchenne Muscular Dystrophy.6 Minute walk test in Duchenne MD patients with different mutations: 12 month changes.Autophagy as a new therapeutic target in Duchenne muscular dystrophy Gene therapy for Duchenne muscular dystrophy: balancing good science, marginal efficacy, high emotions and excessive cost.Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues.Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History.Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping.Myoblots: dystrophin quantification by in-cell western assay for a streamlined development of Duchenne muscular dystrophy (DMD) treatments.The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders.Eteplirsen.Antisense oligonucleotides in neurological disorders.

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P2860

Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy

http://www.wikidata.org/entity/Q35750055

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2011 nî lūn-bûn

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Molecular Therapy

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Restoration of the dystrophin- ...... in Duchenne muscular dystrophy

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Caroline Sewry

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Francesco Muntoni

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Jennifer E Morgan

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Lucy Feng

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Sebahattin Cirak

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Silvia Torelli

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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

Dystrobrevin and dystrophin: an interaction through coiled-coil motifs

ZZ domain of dystrophin and utrophin: topology and mapping of a beta-dystroglycan interaction site

Primary structure of dystrophin-associated glycoproteins linking dystrophin to the extracellular matrix

Differential association of syntrophin pairs with the dystrophin complex

Dystrophin and utrophin influence fiber type composition and post-synaptic membrane structure.

Deletions in the 5' region of dystrophin and resulting phenotypes.

Golgi and sarcolemmal neuronal NOS differentially regulate contraction-induced fatigue and vasoconstriction in exercising mouse skeletal muscle

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

Deficiency of dystrophin-associated proteins in Duchenne muscular dystrophy patients lacking COOH-terminal domains of dystrophin.

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462-467

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10.1038/MT.2011.248

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2

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20

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Virginia Arechavala-Gomeza

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2011-11-15T00:00:00Z

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51801538

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22086232

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3277241

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