Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
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Drug treatment of Duchenne muscular dystrophy: available evidence and perspectivesPre-clinical drug tests in the mdx mouse as a model of dystrophinopathies: an overviewA TALEN-Exon Skipping Design for a Bethlem Myopathy Model in ZebrafishGalectin-1 Protein Therapy Prevents Pathology and Improves Muscle Function in the mdx Mouse Model of Duchenne Muscular DystrophyRecent advances using zebrafish animal models for muscle disease drug discoveryCombined isosorbide dinitrate and ibuprofen as a novel therapy for muscular dystrophies: evidence from Phase I studies in healthy volunteers.Gene and cell-mediated therapies for muscular dystrophy.Proposal for new European pharmaceutical legislation to permit access to custom-made anti-sense oligonucleotide medicinal products.Skeletal muscle homeostasis in duchenne muscular dystrophy: modulating autophagy as a promising therapeutic strategy.New trends in aminoglycosides use.Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubesBlunted cardiac beta-adrenergic response as an early indication of cardiac dysfunction in Duchenne muscular dystrophy.The different impact of a high fat diet on dystrophic mdx and control C57Bl/10 miceMultiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients.UtroUp is a novel six zinc finger artificial transcription factor that recognises 18 base pairs of the utrophin promoter and efficiently drives utrophin upregulation.Improving recognition of Duchenne muscular dystrophy: a retrospective case note reviewProof of concept of the ability of the kinect to quantify upper extremity function in dystrophinopathyPhase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.Application of CRISPR/Cas9 for biomedical discoveries.Fetal skeletal muscle progenitors have regenerative capacity after intramuscular engraftment in dystrophin deficient mice.Longitudinal measurements of MRI-T2 in boys with Duchenne muscular dystrophy: effects of age and disease progressionSkeletal muscle fibrosis in the mdx/utrn+/- mouse validates its suitability as a murine model of Duchenne muscular dystrophy.Targeted excision of VCP R155H mutation by Cre-LoxP technology as a promising therapeutic strategy for valosin-containing protein disease.Recombinase-mediated reprogramming and dystrophin gene addition in mdx mouse induced pluripotent stem cells.Herpes simplex viral-vector design for efficient transduction of nonneuronal cells without cytotoxicity.In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophyDisease course in mdx:utrophin+/- mice: comparison of three mouse models of Duchenne muscular dystrophyEndpoint measures in the mdx mouse relevant for muscular dystrophy pre-clinical studies.Expanding rare disease drug trials based on shared molecular etiologyIdentification in GRMD dog muscle of critical miRNAs involved in pathophysiology and effects associated with MuStem cell transplantation.Nitric oxide sustains long-term skeletal muscle regeneration by regulating fate of satellite cells via signaling pathways requiring Vangl2 and cyclic GMP.Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.Zebrafish models flex their muscles to shed light on muscular dystrophies.Wnt7a treatment ameliorates muscular dystrophy.Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease.Satellite Cells in Muscular Dystrophy - Lost in Polarity.Mutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice.Levels of α7 integrin and laminin-α2 are increased following prednisone treatment in the mdx mouse and GRMD dog models of Duchenne muscular dystrophy.Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.
P2860
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P2860
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
description
2011 nî lūn-bûn
@nan
2011 թուականի Ապրիլին հրատարակուած գիտական յօդուած
@hyw
2011 թվականի ապրիլին հրատարակված գիտական հոդված
@hy
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
name
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@ast
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@en
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@nl
type
label
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@ast
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@en
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@nl
prefLabel
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@ast
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@en
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@nl
P2093
P2860
P356
P1433
P1476
Current status of pharmaceutical and genetic therapeutic approaches to treat DMD.
@en
P2093
Christine I Wooddell
Christophe Pichavant
George Dickson
Jacques P Tremblay
Jon A Wolff
Kay E Davies
Paula R Clemens
Shin'ichi Takeda
Steve D Wilton
P2860
P304
P356
10.1038/MT.2011.59
P577
2011-04-05T00:00:00Z