Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms.
about
Epigenetic associations in relation to cardiovascular prevention and therapeuticsSplicing therapy for neuromuscular diseaseUsing microRNA as an alternative treatment for hyperlipidemia and cardiovascular disease: cardio-miRs in the pipelineTargeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug developmentLong-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension StudyTargeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor.Design and evaluation of locked nucleic acid-based splice-switching oligonucleotides in vitro.Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy.Antisense-mediated Exon Skipping Decreases Tau Protein Expression: A Potential Therapy For TauopathiesComprehensive analysis of tobacco pollen transcriptome unveils common pathways in polar cell expansion and underlying heterochronic shift during spermatogenesis.Therapeutic exon skipping for dysferlinopathies?In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.Regulation of the mutually exclusive exons 8a and 8 in the CaV1.2 calcium channel transcript by polypyrimidine tract-binding protein.Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressivaHuman Splicing Finder: an online bioinformatics tool to predict splicing signals.In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophyAON-mediated Exon Skipping Restores Ciliation in Fibroblasts Harboring the Common Leber Congenital Amaurosis CEP290 Mutation.A prospective study in the rational design of efficient antisense oligonucleotides for exon skipping in the DMD gene.Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP).Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy.Opportunities and challenges for the development of antisense treatment in neuromuscular disorders.Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain.Nucleic-acid based gene therapeutics: delivery challenges and modular design of nonviral gene carriers and expression cassettes to overcome intracellular barriers for sustained targeted expression.Challenges posed to the European pharmaceutical regulatory system by highly personalized medicines.Role of pseudoexons and pseudointrons in human cancer.Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3.Managing the sequence-specificity of antisense oligonucleotides in drug discovery.Antisense Oligonucleotide (AON)-based Therapy for Leber Congenital Amaurosis Caused by a Frequent Mutation in CEP290.Optimization of peptide nucleic acid antisense oligonucleotides for local and systemic dystrophin splice correction in the mdx mouse.A novel dual lock method for down-regulation of genes, in which a target mRNA is captured at 2 independent positions by linked locked nucleic acid antisense oligonucleotides.A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.Intravitreal Injection of Splice-switching Oligonucleotides to Manipulate Splicing in Retinal Cells.Correcting the NLRP3 inflammasome deficiency in macrophages from autoimmune NZB mice with exon skipping antisense oligonucleotides.Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes.From Cryptic Toward Canonical Pre-mRNA Splicing in Pompe Disease: a Pipeline for the Development of Antisense Oligonucleotides.Therapeutic miRNA and siRNA: Moving from Bench to Clinic as Next Generation Medicine.Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 MiceAntisense Oligonucleotide Mediated Splice Correction of a Deep Intronic Mutation in OPA1.Novel Ex Vivo Culture Method for the Study of Dupuytren's Disease: Effects of TGFβ Type 1 Receptor Modulation by Antisense OligonucleotidesPrednisolone treatment does not interfere with 2'-O-methyl phosphorothioate antisense-mediated exon skipping in Duchenne muscular dystrophy
P2860
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P2860
Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms.
description
2008 nî lūn-bûn
@nan
2008年の論文
@ja
2008年学术文章
@wuu
2008年学术文章
@zh-cn
2008年学术文章
@zh-hans
2008年学术文章
@zh-my
2008年学术文章
@zh-sg
2008年學術文章
@yue
2008年學術文章
@zh
2008年學術文章
@zh-hant
name
Guidelines for antisense oligo ...... splice-modulating mechanisms.
@en
type
label
Guidelines for antisense oligo ...... splice-modulating mechanisms.
@en
prefLabel
Guidelines for antisense oligo ...... splice-modulating mechanisms.
@en
P2093
P2860
P356
P1433
P1476
Guidelines for antisense oligo ...... splice-modulating mechanisms.
@en
P2093
Anneke A M Janson
Christa L de Winter
Hans Heemskerk
Judith C T van Deutekom
Laura van Vliet
Marscha Hirschi
Peter A C 't Hoen
Sjef de Kimpe
P2860
P304
P356
10.1038/MT.2008.205
P407
P577
2008-09-23T00:00:00Z