A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground
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Dystrophin-deficient large animal models: translational research and exon skippingThe status of exon skipping as a therapeutic approach to duchenne muscular dystrophyMuscle histology vs MRI in Duchenne muscular dystrophy.Progress in muscular dystrophy research with special emphasis on gene therapy.Duchenne muscular dystrophy gene therapy: Lost in translation?Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 miceDystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trialsAntisense oligo-mediated multiple exon skipping in a dog model of duchenne muscular dystrophy.Transcript expression levels of full-length alpha-synuclein and its three alternatively spliced variants in Parkinson's disease brain regions and in a transgenic mouse model of alpha-synuclein overexpression.Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).Emerging strategies for cell and gene therapy of the muscular dystrophiesMutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice.Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy.Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophyEteplirsen in the treatment of Duchenne muscular dystrophyExon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?New developments in exon skipping and splice modulation therapies for neuromuscular diseases.Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice.More than baby steps: perspectives on pediatric translational research.Highlights from the functional single nucleotide polymorphisms associated with human muscle size and strength or FAMuSS studyEfficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs.Making sense of antisense oligonucleotides: A narrative review.Designing Effective Antisense Oligonucleotides for Exon Skipping.Muscle fat-fraction and mapping in Duchenne muscular dystrophy: evaluation of disease distribution and correlation with clinical assessments. Preliminary experience.Skipping Multiple Exons to Treat DMD-Promises and Challenges.Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy.Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.
P2860
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P2860
A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground
description
2009 nî lūn-bûn
@nan
2009 թուականի Յունուարին հրատարակուած գիտական յօդուած
@hyw
2009 թվականի հունվարին հրատարակված գիտական հոդված
@hy
2009年の論文
@ja
2009年論文
@yue
2009年論文
@zh-hant
2009年論文
@zh-hk
2009年論文
@zh-mo
2009年論文
@zh-tw
2009年论文
@wuu
name
A renaissance for antisense ol ...... xon skipping breaks new ground
@ast
A renaissance for antisense ol ...... xon skipping breaks new ground
@en
type
label
A renaissance for antisense ol ...... xon skipping breaks new ground
@ast
A renaissance for antisense ol ...... xon skipping breaks new ground
@en
prefLabel
A renaissance for antisense ol ...... xon skipping breaks new ground
@ast
A renaissance for antisense ol ...... xon skipping breaks new ground
@en
P2093
P2860
P1433
P1476
A renaissance for antisense ol ...... xon skipping breaks new ground
@en
P2093
Akinori Nakamura
Eric P Hoffman
Qi-Long Lu
Shin'ichi Takeda
Terence A Partridge
Toshifumi Yokota
P2860
P356
10.1001/ARCHNEUROL.2008.540
P577
2009-01-01T00:00:00Z