Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications
about
The pathobiology of splicingLocal restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept studyOveractive bone morphogenetic protein signaling in heterotopic ossification and Duchenne muscular dystrophyNanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophyPotential molecular targeting of splice variants for cancer treatmentSplicing therapy for neuromuscular diseaseEarly pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells.Progress toward therapy with antisense-mediated splicing modulationDoxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettesRescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in miceAntisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patientSilencing disease genes in the laboratory and the clinicEfficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method.CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx MiceNanopolymers 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 dystrophyA duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping.Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor.Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouseGene and cell-mediated therapies for muscular dystrophy.Optimal antisense target reducing INS intron 1 retention is adjacent to a parallel G quadruplex.Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene.Interplay between DMD point mutations and splicing signals in Dystrophinopathy phenotypes.Repair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy.Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressivaBi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy.Modeling the human MTM1 p.R69C mutation in murine Mtm1 results in exon 4 skipping and a less severe myotubular myopathy phenotypeExpanding the action of duplex RNAs into the nucleus: redirecting alternative splicing.Argonaute and the nuclear RNAs: new pathways for RNA-mediated control of gene expression.A prospective study in the rational design of efficient antisense oligonucleotides for exon skipping in the DMD gene.Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP).Allele-specific silencing of the dominant disease allele in sialuria by RNA interference.Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle.Severe acute respiratory syndrome coronavirus triggers apoptosis via protein kinase R but is resistant to its antiviral activityAn endogenous TNF-alpha antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models.Modification of HER2 pre-mRNA alternative splicing and its effects on breast cancer cells.Antisense, RNAi, and gene silencing strategies for therapy: mission possible or impossible?Alternative splicing and disease.Peptide-mediated cellular delivery of oligonucleotide-based therapeutics in vitro: quantitative evaluation of overall efficacy employing easy to handle reporter systems.
P2860
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P2860
Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications
description
2007 nî lūn-bûn
@nan
2007 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2007 թվականի հոտեմբերին հրատարակված գիտական հոդված
@hy
2007年の論文
@ja
2007年論文
@yue
2007年論文
@zh-hant
2007年論文
@zh-hk
2007年論文
@zh-mo
2007年論文
@zh-tw
2007年论文
@wuu
name
Antisense-mediated exon skippi ...... utic and research applications
@ast
Antisense-mediated exon skippi ...... utic and research applications
@en
Antisense-mediated exon skippi ...... utic and research applications
@nl
type
label
Antisense-mediated exon skippi ...... utic and research applications
@ast
Antisense-mediated exon skippi ...... utic and research applications
@en
Antisense-mediated exon skippi ...... utic and research applications
@nl
prefLabel
Antisense-mediated exon skippi ...... utic and research applications
@ast
Antisense-mediated exon skippi ...... utic and research applications
@en
Antisense-mediated exon skippi ...... utic and research applications
@nl
P2860
P3181
P356
P1433
P1476
Antisense-mediated exon skippi ...... utic and research applications
@en
P2860
P304
P3181
P356
10.1261/RNA.653607
P407
P577
2007-10-01T00:00:00Z