Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model.
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Therapeutic Oligonucleotides Targeting Liver Disease: TTR AmyloidosisSplicing therapy for neuromuscular diseaseTargeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug developmentTherapeutic Potential of Tricyclo-DNA antisense oligonucleotidesEvaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular DystrophyTargeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor.Delivery is key: lessons learnt from developing splice-switching antisense therapiesIntracellular Distribution and Nuclear Activity of Antisense Oligonucleotides After Unassisted Uptake in Myoblasts and Differentiated Myotubes In Vitro.RNA therapeutics: beyond RNA interference and antisense oligonucleotides.In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse.Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain.Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.Antisense therapy in neurologyCurrent 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.Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy.Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.X-Linked Dilated Cardiomyopathy: A Cardiospecific Phenotype of Dystrophinopathy.How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouseCholesterol-lowering Action of BNA-based Antisense Oligonucleotides Targeting PCSK9 in Atherogenic Diet-induced Hypercholesterolemic MiceEvolution of thermophilic DNA polymerases for the recognition and amplification of C2'-modified DNA.Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophyCirculating Biomarkers for Duchenne Muscular Dystrophy.New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.Antisense drug discovery and development.Opportunities and challenges for the development of antisense treatment in neuromuscular disorders.Targeting RNA to treat neuromuscular disease.Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain.Cell-matrix interactions in muscle disease.Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy.Oligonucleotides for upregulating gene expression.New developments in exon skipping and splice modulation therapies for neuromuscular diseases.Antisense oligonucleotide therapy for the treatment of C9ORF72 ALS/FTD diseases.Development of therapeutic splice-switching oligonucleotides.The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice.Analysis of the functional consequences of targeted exon deletion in COL7A1 reveals prospects for dystrophic epidermolysis bullosa therapy.Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.Silencing of Foxp3 enhances the antitumor efficacy of GM-CSF genetically modified tumor cell vaccine against B16 melanoma.Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice.AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.
P2860
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P2860
Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model.
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-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
2010年學術文章
@zh-hant
name
Preclinical PK and PD studies ...... otides in the mdx mouse model.
@en
Preclinical PK and PD studies ...... otides in the mdx mouse model.
@nl
type
label
Preclinical PK and PD studies ...... otides in the mdx mouse model.
@en
Preclinical PK and PD studies ...... otides in the mdx mouse model.
@nl
prefLabel
Preclinical PK and PD studies ...... otides in the mdx mouse model.
@en
Preclinical PK and PD studies ...... otides in the mdx mouse model.
@nl
P2093
P2860
P356
P1433
P1476
Preclinical PK and PD studies ...... otides in the mdx mouse model.
@en
P2093
Alessandra Ferlini
Christa de Winter
Hans Heemskerk
Judith C T van Deutekom
Niki Heuvelmans
Paola Braghetta
Paola Rimessi
Patrizia Sabatelli
Petra van Kuik
Sjef de Kimpe
P2860
P304
P356
10.1038/MT.2010.72
P577
2010-04-20T00:00:00Z