Targeted exon skipping to correct exon duplications in the dystrophin gene. - Wikidata - wikimedia - TriplyDB

Targeted exon skipping to correct exon duplications in the dystrophin gene.

Targeted exon skipping to correct exon duplications in the dystrophin gene.

http://www.wikidata.org/entity/Q37695543

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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.

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P2860

Targeted exon skipping to correct exon duplications in the dystrophin gene.

http://www.wikidata.org/entity/Q37695543

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

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Molecular therapy. Nucleic acids

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Targeted exon skipping to correct exon duplications in the dystrophin gene

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Hanns Lochmüller

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Kane L Greer

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Steve D Wilton

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P2860

Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries

The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein

ESEfinder: A web resource to identify exonic splicing enhancers

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy

Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations.

Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule.

The influence of antisense oligonucleotide length on dystrophin exon skipping.

Induction of revertant fibres in the mdx mouse using antisense oligonucleotides

Targeted exon skipping to address "leaky" mutations in the dystrophin gene.

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e155

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scholarly article

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10.1038/MTNA.2014.8

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3

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Kevin M. Flanigan

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2014-03-18T00:00:00Z

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260915980

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