Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.
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A chemical view of oligonucleotides for exon skipping and related drug applicationsAntisense oligonucleotides: treating neurodegeneration at the level of RNASplicing therapy for neuromuscular diseaseCurrent understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophyAnimal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyMYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correctionIdentification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screeningSilencing disease genes in the laboratory and the clinicPrevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.Dual suppression of hemangiogenesis and lymphangiogenesis by splice-shifting morpholinos targeting vascular endothelial growth factor receptor 2 (KDR)Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy.Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping EfficiencyCell penetrating peptides in the delivery of biopharmaceuticals.Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouseOcta-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice.Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular AtrophyGene and cell-mediated therapies for muscular dystrophy.Dystrophin isoform induction in vivo by antisense-mediated alternative splicing.Gene knockdowns in adult animals: PPMOs and vivo-morpholinos.Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching.Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia modelExon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko miceRNA therapeutics: beyond RNA interference and antisense oligonucleotides.Anti-tumor activity of splice-switching oligonucleotides.Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe DiseasePhysiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy.The status of exon skipping as a therapeutic approach to duchenne muscular dystrophyMammalian models of Duchenne Muscular Dystrophy: pathological characteristics and therapeutic applications.Experimental models of duchenne muscular dystrophy: relationship with cardiovascular disease.Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.Inhibition of nonsense-mediated mRNA decay by antisense morpholino oligonucleotides restores functional expression of hERG nonsense and frameshift mutations in long-QT syndromeEfficient in vivo manipulation of alternative pre-mRNA splicing events using antisense morpholinos in mice.Regulation of the mutually exclusive exons 8a and 8 in the CaV1.2 calcium channel transcript by polypyrimidine tract-binding protein.Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy.Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.Pip5 transduction peptides direct high efficiency oligonucleotide-mediated dystrophin exon skipping in heart and phenotypic correction in mdx mice.Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum.
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P2860
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.
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article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on 10 June 2008
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Sustained dystrophin expressio ...... rs in the muscles of mdx mice.
@en
Sustained dystrophin expressio ...... rs in the muscles of mdx mice.
@nl
type
label
Sustained dystrophin expressio ...... rs in the muscles of mdx mice.
@en
Sustained dystrophin expressio ...... rs in the muscles of mdx mice.
@nl
prefLabel
Sustained dystrophin expressio ...... rs in the muscles of mdx mice.
@en
Sustained dystrophin expressio ...... rs in the muscles of mdx mice.
@nl
P2093
P2860
P356
P1433
P1476
Sustained dystrophin expressio ...... ers in the muscles of mdx mice
@en
P2093
Brian Buckley
Hong M Moulton
Jennifer Roberts
Natee Jearawiriyapaisarn
Peter Sazani
Suthat Fucharoen
P2860
P304
P356
10.1038/MT.2008.120
P577
2008-06-10T00:00:00Z