Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping.
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Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.Proteasomal inhibition restores biological function of mis-sense mutated dysferlin in patient-derived muscle cells.Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.Modular dispensability of dysferlin C2 domains reveals rational design for mini-dysferlin molecules.Therapeutic exon skipping for dysferlinopathies?Antisense therapy in neurologyProgress in muscular dystrophy research with special emphasis on gene therapy.Myofiber damage precedes macrophage infiltration after in vivo injury in dysferlin-deficient A/J mouse skeletal muscle.Dysferlinopathy Fibroblasts Are Defective in Plasma Membrane Repair.Targeting RNA splicing for disease therapy.Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy.Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy.Full-length Dysferlin Transfer by the Hyperactive Sleeping Beauty Transposase Restores Dysferlin-deficient Muscle.Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients' CellsHip region muscular dystrophy and emergence of motor deficits in dysferlin-deficient Bla/J mice.New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.Current advances in cell therapy strategies for muscular dystrophies.Opportunities and challenges for the development of antisense treatment in neuromuscular disorders.Translational research and therapeutic perspectives in dysferlinopathies.Generation of skeletal muscle cells from embryonic and induced pluripotent stem cells as an in vitro model and for therapy of muscular dystrophies.Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies.New developments in exon skipping and splice modulation therapies for neuromuscular diseases.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.In vitro correction of a pseudoexon-generating deep intronic mutation in LGMD2A by antisense oligonucleotides and modified small nuclear RNAs.Identification of splicing defects caused by mutations in the dysferlin gene.Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3' splice sites.UMD-DYSF, a novel locus specific database for the compilation and interactive analysis of mutations in the dysferlin gene.Identification of variants in the 4q35 gene FAT1 in patients with a facioscapulohumeral dystrophy-like phenotype.A novel dysferlin mutant pseudoexon bypassed with antisense oligonucleotides.Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells.Therapeutic exon 'switching' for dysferlinopathies?Dysferlin aggregation in limb-girdle muscular dystrophy type 2B/Miyoshi Myopathy necessitates mutational screen for diagnosis [corrected].A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathy.Efficient Skipping of Single Exon Duplications in DMD Patient-Derived Cell Lines Using an Antisense Oligonucleotide Approach.Designing Effective Antisense Oligonucleotides for Exon Skipping.RETRACTED: Proteasome inhibitors increase missense mutated dysferlin in patients with muscular dystrophy.RNA Editing for Muscular Dystrophy TherapyIdentification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy
P2860
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P2860
Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping.
description
2010 nî lūn-bûn
@nan
2010年の論文
@ja
2010年学术文章
@wuu
2010年学术文章
@zh
2010年学术文章
@zh-cn
2010年学术文章
@zh-hans
2010年学术文章
@zh-my
2010年学术文章
@zh-sg
2010年學術文章
@yue
2010年學術文章
@zh-hant
name
Efficient bypass of mutations ...... tisense-induced exon skipping.
@en
Efficient bypass of mutations ...... tisense-induced exon skipping.
@nl
type
label
Efficient bypass of mutations ...... tisense-induced exon skipping.
@en
Efficient bypass of mutations ...... tisense-induced exon skipping.
@nl
prefLabel
Efficient bypass of mutations ...... tisense-induced exon skipping.
@en
Efficient bypass of mutations ...... tisense-induced exon skipping.
@nl
P2093
P50
P356
P1433
P1476
Efficient bypass of mutations ...... tisense-induced exon skipping.
@en
P2093
Anthony Behin
Aurélie Avril
Cyriaque Beley
Luis Garcia
Martin Krahn
Nicolas Wein
Pascal Laforêt
Soraya Chaouch
P304
P356
10.1002/HUMU.21160
P577
2010-02-01T00:00:00Z