Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells.
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Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapyGene therapy in clinical medicineAntisense-mediated exon skipping: a versatile tool with therapeutic and research applicationsSplicing therapy for neuromuscular diseaseWhat can Duchenne Connect teach us about treating Duchenne muscular dystrophy?Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in miceInvestigating Synthetic Oligonucleotide Targeting of Mir31 in Duchenne Muscular DystrophyOligonucleotides and derivatives as gene-specific control agents.Antisense-induced exon skipping for duplications in Duchenne muscular dystrophyDynamics of co-transcriptional pre-mRNA folding influences the induction of dystrophin exon skipping by antisense oligonucleotides.Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophyGenetic correction of splice site mutation in purified and enriched myoblasts isolated from mdx5cv mice.Modulation of mdm2 pre-mRNA splicing by 9-aminoacridine-PNA (peptide nucleic acid) conjugates targeting intron-exon junctions.Producing recombinant adeno-associated virus in foster cells: overcoming production limitations using a baculovirus-insect cell expression strategy.Gene and cell-mediated therapies for muscular dystrophy.Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cellsRNA therapeutics: beyond RNA interference and antisense oligonucleotides.Therapeutic exon skipping for dysferlinopathies?In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.Antisense therapy in neurologyInduction of revertant fibres in the mdx mouse using antisense oligonucleotidesEffective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient miceHigh throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomicsRecurrent deep intronic mutations in the SLC12A3 gene responsible for Gitelman's syndromeCurrent status of pharmaceutical and genetic therapeutic approaches to treat DMD.Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing.Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish.Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.Natural repair mechanisms in correcting pathogenic mutations in inherited skin disorders.Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides.Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skippingViral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advancesDigital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy.Focal but reversible diastolic sheet dysfunction reflects regional calcium mishandling in dystrophic mdx mouse hearts.Molecular therapeutic strategies targeting Duchenne muscular dystrophyEmerging strategies for cell and gene therapy of the muscular dystrophiesThe potential of exon skipping for treatment for Duchenne muscular dystrophy.Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy
P2860
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P2860
Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells.
description
2001 nî lūn-bûn
@nan
2001年の論文
@ja
2001年学术文章
@wuu
2001年学术文章
@zh-cn
2001年学术文章
@zh-hans
2001年学术文章
@zh-my
2001年学术文章
@zh-sg
2001年學術文章
@yue
2001年學術文章
@zh
2001年學術文章
@zh-hant
name
Antisense-induced exon skippin ...... patient derived muscle cells.
@en
type
label
Antisense-induced exon skippin ...... patient derived muscle cells.
@en
prefLabel
Antisense-induced exon skippin ...... patient derived muscle cells.
@en
P2093
P356
P1476
Antisense-induced exon skippin ...... patient derived muscle cells.
@en
P2093
Bremmer-Bout M
Ginjaar IB
den Dunnen JT
van Deutekom JC
van Ommen GJ
P304
P356
10.1093/HMG/10.15.1547
P577
2001-07-01T00:00:00Z