Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy.
about
The potential of sarcospan in adhesion complex replacement therapeutics for the treatment of muscular dystrophyThe ubiquitin proteasome system in human cardiomyopathies and heart failure.High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomicsEffects of Dantrolene Therapy on Disease Phenotype in Dystrophin Deficient mdx Mice.Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy.Reengineering a transmembrane protein to treat muscular dystrophy using exon skippingHexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice.The small molecule Retro-1 enhances the pharmacological actions of antisense and splice switching oligonucleotides.Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy.The delivery of therapeutic oligonucleotidesTherapeutic advances in muscular dystrophyCellular uptake and intracellular trafficking of oligonucleotides: implications for oligonucleotide pharmacology.Ongoing therapeutic trials and outcome measures for Duchenne muscular dystrophy.Role of pseudoexons and pseudointrons in human cancer.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Cellular uptake and intracellular trafficking of oligonucleotidesDrug Discovery of Therapies for Duchenne Muscular Dystrophy.RNA mis-splicing in disease.Translational development of splice-modifying antisense oligomers.2'-O-Methyl RNA/Ethylene-Bridged Nucleic Acid Chimera Antisense Oligonucleotides to Induce Dystrophin Exon 45 Skipping.Modulation of aberrant splicing in human RNA diseases by chemical compounds.Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy.A novel dysferlin mutant pseudoexon bypassed with antisense oligonucleotides.Tween 85-Modified Low Molecular Weight PEI Enhances Exon-Skipping of Antisense Morpholino Oligomer In Vitro and in mdx Mice.Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice.Direct reprogramming of urine-derived cells with inducible MyoD for modeling human muscle disease.Combined Therapies for Duchenne Muscular Dystrophy to Optimize Treatment Efficacy.Ammonium and arsenic trioxide are potent facilitators of oligonucleotide function when delivered by gymnosis.Treating pediatric neuromuscular disorders: The future is now.PAX3-FOXO1 transgenic zebrafish models identify HES3 as a mediator of rhabdomyosarcoma tumorigenesis.Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse.Saponins as Natural Adjuvant for Antisense Morpholino Oligonucleotides Delivery In Vitro and in mdx Mice.Multidirectional Efficacy of Biologically Active Nitro Compounds Included in Medicines.Saponins enhance exon skipping of 2′-O-methyl phosphorothioate oligonucleotide in vitro and in vivo
P2860
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P2860
Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy.
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
2012年论文
@zh
2012年论文
@zh-cn
name
Dantrolene enhances antisense- ...... f Duchenne muscular dystrophy.
@en
Dantrolene enhances antisense- ...... f Duchenne muscular dystrophy.
@nl
type
label
Dantrolene enhances antisense- ...... f Duchenne muscular dystrophy.
@en
Dantrolene enhances antisense- ...... f Duchenne muscular dystrophy.
@nl
prefLabel
Dantrolene enhances antisense- ...... f Duchenne muscular dystrophy.
@en
Dantrolene enhances antisense- ...... f Duchenne muscular dystrophy.
@nl
P2093
P2860
P1476
Dantrolene enhances antisense- ...... f Duchenne muscular dystrophy.
@en
P2093
Derek W Wang
Ekaterina I Mokhonova
Genevieve C Kendall
Leonel Martinez
M Carrie Miceli
Melissa J Spencer
Miriana Moran
Natalia E Sejbuk
Oscar Silva
P2860
P304
P356
10.1126/SCITRANSLMED.3005054
P407
P577
2012-12-01T00:00:00Z