Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
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Aminoglycosides and other nonsense suppression therapies for the treatment of dystrophinopathyDrug treatment of Duchenne muscular dystrophy: available evidence and perspectivesExon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation studyLocal restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept studyDystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophiesMarginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout miceDisease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatmentsAdvances in gene therapy for muscular dystrophiesAnimal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyIdentification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screeningLow dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout miceWhat Can We Learn From Clinical Trials of Exon Skipping for DMD?Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.Emerging genetic therapies to treat Duchenne muscular dystrophyForelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.The effects of low levels of dystrophin on mouse muscle function and pathologyThe absence of dystrophin brain isoform expression in healthy human heart ventricles explains the pathogenesis of 5' X-linked dilated cardiomyopathy.Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene.Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophyThe DMD locus harbours multiple long non-coding RNAs which orchestrate and control transcription of muscle dystrophin mRNA isoforms.A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.Physiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy.Enhancing translation: guidelines for standard pre-clinical experiments in mdx miceDystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trialsHow much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouseA prospective study in the rational design of efficient antisense oligonucleotides for exon skipping in the DMD gene.piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.Preservation of muscle force in Mdx3cv mice correlates with low-level expression of a near full-length dystrophin proteinL-arginine decreases inflammation and modulates the nuclear factor-kappaB/matrix metalloproteinase cascade in mdx muscle fibers.Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.In vivo gene editing in dystrophic mouse muscle and muscle stem cells.Designing heart performance by gene transfer.Circulating Biomarkers for Duchenne Muscular Dystrophy.Therapeutics based on stop codon readthrough.Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy.Normal and altered pre-mRNA processing in the DMD gene.
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P2860
Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
description
im Dezember 2007 veröffentlichter wissenschaftlicher Artikel
@de
wetenschappelijk artikel
@nl
наукова стаття, опублікована в грудні 2007
@uk
name
Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
@en
Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
@nl
type
label
Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
@en
Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
@nl
prefLabel
Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
@en
Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
@nl
P2093
P50
P1476
Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human
@en
P2093
Alessandra Ferlini
Caroline Sewry
Francesca Gualandi
Isabella Ugo
Marcella Neri
Paola Rimessi
Patrizia Sabatelli
Silvia Torelli
P304
P356
10.1016/J.NMD.2007.07.005
P577
2007-12-01T00:00:00Z