Targeted exon skipping to correct exon duplications in the dystrophin gene.
about
Gene therapy for the nervous system: challenges and new strategies.Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies.Exon skipping: a first in class strategy for Duchenne muscular dystrophy.Comparative mass spectrometric and immunoassay-based proteome analysis in serum of Duchenne muscular dystrophy patients.Health care utilization and costs for children and adults with duchenne muscular dystrophy.Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach.Improved Diagnosis and Care for Rare Diseases through Implementation of Precision Public Health Framework.The potential of antisense oligonucleotide therapies for inherited childhood lung diseases.Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy.
P2860
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P2860
Targeted exon skipping to correct exon duplications in the dystrophin gene.
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 18 March 2014
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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
Targeted exon skipping to correct exon duplications in the dystrophin gene.
@en
Targeted exon skipping to correct exon duplications in the dystrophin gene.
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type
label
Targeted exon skipping to correct exon duplications in the dystrophin gene.
@en
Targeted exon skipping to correct exon duplications in the dystrophin gene.
@nl
prefLabel
Targeted exon skipping to correct exon duplications in the dystrophin gene.
@en
Targeted exon skipping to correct exon duplications in the dystrophin gene.
@nl
P2093
P2860
P356
P1476
Targeted exon skipping to correct exon duplications in the dystrophin gene
@en
P2093
Hanns Lochmüller
Kane L Greer
Steve D Wilton
P2860
P356
10.1038/MTNA.2014.8
P577
2014-03-18T00:00:00Z