New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
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Splicing therapy for neuromuscular diseaseThe effects of low levels of dystrophin on mouse muscle function and pathologySuccessful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies.Deep-intronic ATM mutation detected by genomic resequencing and corrected in vitro by antisense morpholino oligonucleotide (AMO).In vitro antisense therapeutics for a deep intronic mutation causing Neurofibromatosis type 2.6 Minute walk test in Duchenne MD patients with different mutations: 12 month changes.Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations.Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle.Challenges of biomarkers in drug discovery and development.Megalencephalic leukoencephalopathy with subcortical cysts type 1 (MLC1) due to a homozygous deep intronic splicing mutation (c.895-226T>G) abrogated in vitro using an antisense morpholino oligonucleotide.
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New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on December 2010
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
@en
New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
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type
label
New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
@en
New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
@nl
prefLabel
New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
@en
New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
@nl
P2860
P1476
New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.
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P2093
Johan T den Dunnen
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10.1111/J.1749-6632.2010.05836.X
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P577
2010-12-01T00:00:00Z