Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy
about
Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skippingEmerging genetic therapies to treat Duchenne muscular dystrophyPersonalized exon skipping strategies to address clustered non-deletion dystrophin mutations.Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy.Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 miceClinical phenotypes as predictors of the outcome of skipping around DMD exon 45.Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trialsBodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophyGene therapy for muscular dystrophy: current progress and future prospects.New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?Exon skipping: a first in class strategy for Duchenne muscular dystrophy.Normal and altered pre-mRNA processing in the DMD gene.Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.Differential stabilities of alternative exon-skipped rod motifs of dystrophin.Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.Antisense mediated exon skipping therapy for duchenne muscular dystrophy (DMD)Reply to Lévy et al.Making sense of antisense oligonucleotides: A narrative review.Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.
P2860
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P2860
Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy
description
2008 nî lūn-bûn
@nan
2008 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2008 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2008年の論文
@ja
2008年論文
@yue
2008年論文
@zh-hant
2008年論文
@zh-hk
2008年論文
@zh-mo
2008年論文
@zh-tw
2008年论文
@wuu
name
Assessment of the feasibility ...... or Duchenne muscular dystrophy
@ast
Assessment of the feasibility ...... or Duchenne muscular dystrophy
@en
type
label
Assessment of the feasibility ...... or Duchenne muscular dystrophy
@ast
Assessment of the feasibility ...... or Duchenne muscular dystrophy
@en
prefLabel
Assessment of the feasibility ...... or Duchenne muscular dystrophy
@ast
Assessment of the feasibility ...... or Duchenne muscular dystrophy
@en
P2093
P2860
P356
P1433
P1476
Assessment of the feasibility ...... or Duchenne muscular dystrophy
@en
P2093
Christa L de Winter
Judith C T van Deutekom
Laura van Vliet
P2860
P2888
P356
10.1186/1471-2350-9-105
P577
2008-12-01T00:00:00Z
P5875
P6179
1043588432