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Lack of correlation between outcomes of membrane repair assay and correction of dystrophic changes in experimental therapeutic strategy in dysferlinopathyProteasomal inhibition restores biological function of mis-sense mutated dysferlin in patient-derived muscle cells.Modular dispensability of dysferlin C2 domains reveals rational design for mini-dysferlin molecules.Antisense therapy in neurologyInhibition of inflammation with celastrol fails to improve muscle function in dysferlin-deficient A/J miceProspect of gene therapy for cardiomyopathy in hereditary muscular dystrophy.Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients' CellsOpportunities and challenges for the development of antisense treatment in neuromuscular disorders.Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?UMD-DYSF, a novel locus specific database for the compilation and interactive analysis of mutations in the dysferlin gene.A novel dysferlin mutant pseudoexon bypassed with antisense oligonucleotides.Gene therapy matures in the clinic.RETRACTED: Proteasome inhibitors increase missense mutated dysferlin in patients with muscular dystrophy.Abnormal expression of dysferlin in skeletal muscle and monocytes supports primary dysferlinopathy in patients with one mutated allele.
P2860
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P2860
description
2010 nî lūn-bûn
@nan
2010 թուականի Փետրուարին հրատարակուած գիտական յօդուած
@hyw
2010 թվականի փետրվարին հրատարակված գիտական հոդված
@hy
2010年の論文
@ja
2010年論文
@yue
2010年論文
@zh-hant
2010年論文
@zh-hk
2010年論文
@zh-mo
2010年論文
@zh-tw
2010年论文
@wuu
name
Therapeutic exon skipping for dysferlinopathies?
@ast
Therapeutic exon skipping for dysferlinopathies?
@en
Therapeutic exon skipping for dysferlinopathies?
@nl
type
label
Therapeutic exon skipping for dysferlinopathies?
@ast
Therapeutic exon skipping for dysferlinopathies?
@en
Therapeutic exon skipping for dysferlinopathies?
@nl
prefLabel
Therapeutic exon skipping for dysferlinopathies?
@ast
Therapeutic exon skipping for dysferlinopathies?
@en
Therapeutic exon skipping for dysferlinopathies?
@nl
P2093
P2860
P50
P356
P1476
Therapeutic exon skipping for dysferlinopathies?
@en
P2093
Gert-Jan van Ommen
Ieke B Ginjaar
Ivo F A C Fokkema
Johan T den Dunnen
Kavita H K Singh
P2860
P2888
P304
P356
10.1038/EJHG.2010.4
P577
2010-02-10T00:00:00Z