Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells.
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Antisense-mediated exon skipping: a versatile tool with therapeutic and research applicationsA chemical view of oligonucleotides for exon skipping and related drug applicationsAntisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistriesNanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice.Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophySystemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal musclesDesign and evaluation of locked nucleic acid-based splice-switching oligonucleotides in vitro.Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching.Potent and selective inhibition of A-to-I RNA editing with 2'-O-methyl/locked nucleic acid-containing antisense oligoribonucleotides.A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver.Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain.Gene therapy progress and prospects: Duchenne muscular dystrophy.Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophy.Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressivaShielding the messenger (RNA): microRNA-based anticancer therapies.Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.MiRNA inhibition in tissue engineering and regenerative medicine.Epigenetic manipulation of gene expression: a toolkit for cell biologistsNovel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agentsPeptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice.Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophyTherapeutic potential of splice-switching oligonucleotides.Overcoming biological barriers to in vivo efficacy of antisense oligonucleotides.Exon 32 Skipping of Dysferlin Rescues Membrane Repair in Patients' CellsMolecular beacons and related probes for intracellular RNA imaging.Antisense drug discovery and development.Targeting RNA to treat neuromuscular disease.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases.Development of therapeutic splice-switching oligonucleotides.Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.A triple-helix forming oligonucleotide targeting genomic DNA fails to induce mutation.Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model.Short (16-mer) locked nucleic acid splice-switching oligonucleotides restore dystrophin production in Duchenne Muscular Dystrophy myotubes.Antisense Oligonucleotide Mediated Splice Correction of a Deep Intronic Mutation in OPA1.Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells.Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations.Rational Design of Short Locked Nucleic Acid-Modified 2′-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro.Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD.
P2860
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P2860
Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells.
description
2004 nî lūn-bûn
@nan
2004年の論文
@ja
2004年学术文章
@wuu
2004年学术文章
@zh-cn
2004年学术文章
@zh-hans
2004年学术文章
@zh-my
2004年学术文章
@zh-sg
2004年學術文章
@yue
2004年學術文章
@zh
2004年學術文章
@zh-hant
name
Comparative analysis of antise ...... n 46 skipping in muscle cells.
@en
Comparative analysis of antise ...... n 46 skipping in muscle cells.
@nl
type
label
Comparative analysis of antise ...... n 46 skipping in muscle cells.
@en
Comparative analysis of antise ...... n 46 skipping in muscle cells.
@nl
prefLabel
Comparative analysis of antise ...... n 46 skipping in muscle cells.
@en
Comparative analysis of antise ...... n 46 skipping in muscle cells.
@nl
P2093
P2860
P356
P1433
P1476
Comparative analysis of antise ...... n 46 skipping in muscle cells.
@en
P2093
A A M Janson
A Aartsma-Rus
G-J B van Ommen
J C T van Deutekom
J T den Dunnen
M Bremmer-Bout
P2860
P2888
P304
P356
10.1038/SJ.GT.3302313
P577
2004-09-01T00:00:00Z