Exon skipping-mediated dystrophin reading frame restoration for small mutations

Exon skipping-mediated dystrophin reading frame restoration for small mutations

Item
http://www.wikidata.org/entity/Q57639981
Splicing therapy for neuromuscular diseaseClinical and molecular characterization of a cohort of patients with novel nucleotide alterations of the Dystrophin gene detected by direct sequencingPersonalized exon skipping strategies to address clustered non-deletion dystrophin mutations.The DMD locus harbours multiple long non-coding RNAs which orchestrate and control transcription of muscle dystrophin mRNA isoforms.The status of exon skipping as a therapeutic approach to duchenne muscular dystrophyDuchenne muscular dystrophy gene therapy: Lost in translation?Multiple exon skipping strategies to by-pass dystrophin mutations.Targeting RNA splicing for disease therapy.Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agentsNew insights in gene-derived therapy: the example of Duchenne muscular dystrophy.Pharmacology of Modulators of Alternative Splicing.Improved diagnostic yield of neuromuscular disorders applying clinical exome sequencing in patients arising from a consanguineous population.Persistent dystrophin protein restoration 90 days after a course of intraperitoneally administered naked 2'OMePS AON and ZM2 NP-AON complexes in mdx mice.Biodistribution and molecular studies on orally administered nanoparticle-AON complexes encapsulated with alginate aiming at inducing dystrophin rescue in mdx mice.Antisense-induced messenger depletion corrects a COL6A2 dominant mutation in Ullrich myopathyDiscovery of pathogenic variants in a large Korean cohort of inherited muscular disorders.
P2860

Exon skipping-mediated dystrophin reading frame restoration for small mutations

Item
http://www.wikidata.org/entity/Q57639981
description
im November 2009 veröffentlichter wissenschaftlicher Artikel
@de
wetenschappelijk artikel
@nl
наукова стаття, опублікована в листопаді 2009
@uk
name
Exon skipping-mediated dystrophin reading frame restoration for small mutations
@en
Exon skipping-mediated dystrophin reading frame restoration for small mutations
@nl
type
label
Exon skipping-mediated dystrophin reading frame restoration for small mutations
@en
Exon skipping-mediated dystrophin reading frame restoration for small mutations
@nl
prefLabel
Exon skipping-mediated dystrophin reading frame restoration for small mutations
@en
Exon skipping-mediated dystrophin reading frame restoration for small mutations
@nl
P1433
P1476
P2093
P304
P31
P356
P433
P478
P50
P577
P698
P356
P1433
P1476
Exon skipping-mediated dystrophin reading frame restoration for small mutations
@en
P2093
P304
P31
P356
P433
P478
P50
P577
P698