Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.
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Drug treatment of Duchenne muscular dystrophy: available evidence and perspectivesExon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation studySplicing therapy for neuromuscular diseaseCurrent understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophyRescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skippingPrevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.Dystrophic Cardiomyopathy-Potential Role of Calcium in Pathogenesis, Treatment and Novel TherapiesEvaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx miceCurrent status of pharmaceutical and genetic therapeutic approaches to treat DMD.Dystromirs as serum biomarkers for monitoring the disease severity in Duchenne muscular Dystrophy.Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy.Could exon skipping help dystrophic boys to run, hop, and jump?Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophyHow much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouseMusculoskeletal Geometry, Muscle Architecture and Functional Specialisations of the Mouse HindlimbCationic polyelectrolyte-mediated delivery of antisense morpholino oligonucleotides for exon-skipping in vitro and in mdx mice.Membrane-stabilizing copolymers confer marked protection to dystrophic skeletal muscle in vivo.Elusive sources of variability of dystrophin rescue by exon skippingBehavioral and locomotor measurements using an open field activity monitoring system for skeletal muscle diseases.Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.Circulating Biomarkers for Duchenne Muscular Dystrophy.Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells.Opportunities and challenges for the development of antisense treatment in neuromuscular disorders.Targeting RNA to treat neuromuscular disease.Cell-matrix interactions in muscle disease.Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Drug Discovery of Therapies for Duchenne Muscular Dystrophy.Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy.Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models.Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne.Myoblots: dystrophin quantification by in-cell western assay for a streamlined development of Duchenne muscular dystrophy (DMD) treatments.Ryanodine channel complex stabilizer compound S48168/ARM210 as a disease modifier in dystrophin-deficient mdx mice: proof-of-concept study and independent validation of efficacy.Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice.
P2860
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P2860
Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.
description
2010 nî lūn-bûn
@nan
2010 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած
@hyw
2010 թվականի նոյեմբերին հրատարակված գիտական հոդված
@hy
2010年の論文
@ja
2010年論文
@yue
2010年論文
@zh-hant
2010年論文
@zh-hk
2010年論文
@zh-mo
2010年論文
@zh-tw
2010年论文
@wuu
name
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@ast
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@en
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@nl
type
label
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@ast
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@en
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@nl
prefLabel
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@ast
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@en
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@nl
P2093
P2860
P50
P356
P1433
P1476
Chronic systemic therapy with ...... ocomotor behavior in mdx mice.
@en
P2093
Alberto Malerba
Francesco Muntoni
George Dickson
Ian R Graham
Paul S Sharp
P2860
P304
P356
10.1038/MT.2010.261
P577
2010-11-23T00:00:00Z