Found: 114
Select item for more details and to access through your institution.
Clinical trial network for the promotion of clinical research for rare diseases in Japan: muscular dystrophy clinical trial network.
- Published in:
- 2016
- By:
- Publication type:
- journal article
Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog.
- Published in:
- Journal of Gene Medicine, 2011, v. 13, n. 9, p. 497, doi. 10.1002/jgm.1602
- By:
- Publication type:
- Article
Downstream utrophin enhancer is required for expression of utrophin in skeletal muscle.
- Published in:
- Journal of Gene Medicine, 2008, v. 10, n. 6, p. 702, doi. 10.1002/jgm.1190
- By:
- Publication type:
- Article
The utrophin promoter A drives high expression of the transgenic LacZ gene in liver, testis, colon, submandibular gland, and small intestine.
- Published in:
- Journal of Gene Medicine, 2005, v. 7, n. 2, p. 237, doi. 10.1002/jgm.651
- By:
- Publication type:
- Article
Identification and characterization of a novel human dysferlin transcript: dysferlin_v1.
- Published in:
- Human Genetics, 2006, v. 120, n. 3, p. 410, doi. 10.1007/s00439-006-0230-1
- By:
- Publication type:
- Article
Exon-skipping therapy for Duchenne muscular dystrophy.
- Published in:
- Neuropathology, 2009, v. 29, n. 4, p. 494, doi. 10.1111/j.1440-1789.2009.01028.x
- By:
- Publication type:
- Article
Fukutin is prerequisite to ameliorate muscular dystrophic phenotype by myofiber-selective LARGE expression.
- Published in:
- Scientific Reports, 2015, p. 8316, doi. 10.1038/srep08316
- By:
- Publication type:
- Article
Initial Pulmonary Respiration Causes Massive Diaphragm Damage and Hyper-CKemia in Duchenne Muscular Dystrophy Dog.
- Published in:
- Scientific Reports, 2013, p. 1, doi. 10.1038/srep02183
- By:
- Publication type:
- Article
Reprogramming efficiency and quality of induced Pluripotent Stem Cells (iPSCs) generated from muscle-derived fibroblasts of mdx mice at different ages.
- Published in:
- PLoS Currents, 2011, p. 525, doi. 10.1371/currents.RRN1274
- By:
- Publication type:
- Article
Characterization of a novel microRNA, miR-188, elevated in serum of muscular dystrophy dog model.
- Published in:
- PLoS ONE, 2019, v. 14, n. 01, p. 1, doi. 10.1371/journal.pone.0211597
- By:
- Publication type:
- Article
Accelerometric outcomes of motor function related to clinical evaluations and muscle involvement in dystrophic dogs.
- Published in:
- PLoS ONE, 2018, v. 13, n. 12, p. 1, doi. 10.1371/journal.pone.0208415
- By:
- Publication type:
- Article
Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts.
- Published in:
- PLoS ONE, 2018, v. 13, n. 5, p. 1, doi. 10.1371/journal.pone.0197084
- By:
- Publication type:
- Article
A conserved regulatory element located far downstream of the gls locus modulates gls expression through chromatin loop formation during myogenesis
- Published in:
- FEBS Letters, 2012, v. 586, n. 19, p. 3464, doi. 10.1016/j.febslet.2012.07.074
- By:
- Publication type:
- Article
Generation of transplantable, functional satellite-like cells from mouse embryonic stem cells.
- Published in:
- FASEB Journal, 2009, v. 23, n. 6, p. 1907, doi. 10.1096/fj.08-123661
- By:
- Publication type:
- Article
SynArfGEF is a guanine nucleotide exchange factor for Arf6 and localizes preferentially at post-synaptic specializations of inhibitory synapses.
- Published in:
- Journal of Neurochemistry, 2011, v. 116, n. 6, p. 1122, doi. 10.1111/j.1471-4159.2010.07167.x
- By:
- Publication type:
- Article
Intra-Amniotic rAAV-Mediated Microdystrophin Gene Transfer Improves Canine X-Linked Muscular Dystrophy and May Induce Immune Tolerance.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 4, p. 627, doi. 10.1038/mt.2015.5
- By:
- Publication type:
- Article
Cardiac side population cells have a potential to migrate and differentiate into cardiomyocytes in vitro and in viva.
- Published in:
- Journal of Cell Biology, 2007, v. 176, n. 3, p. 329, doi. 10.1083/jcb.200603014
- By:
- Publication type:
- Article
Coordinate control of axon defasciculation and myelination by laminin-2 and -8.
- Published in:
- Journal of Cell Biology, 2005, v. 168, n. 4, p. 655, doi. 10.1083/jcb.200411158
- By:
- Publication type:
- Article
α-Syntrophin-deficient skeletal muscle exhibits hypertrophy and aberrant formation of neuromuscular junctions during regeneration.
- Published in:
- Journal of Cell Biology, 2002, v. 158, n. 6, p. 1097, doi. 10.1083/jcb.200204076
- By:
- Publication type:
- Article
Characterization of deletion breakpoints in patients with dystrophinopathy carrying a deletion of exons 45–55 of the Duchenne muscular dystrophy (DMD) gene.
- Published in:
- Journal of Human Genetics, 2009, v. 54, n. 2, p. 127, doi. 10.1038/jhg.2008.8
- By:
- Publication type:
- Article
Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients.
- Published in:
- Human Molecular Genetics, 2014, v. 23, n. 24, p. 6458, doi. 10.1093/hmg/ddu366
- By:
- Publication type:
- Article
Dystrophic mdx mice develop severe cardiac and respiratory dysfunction following genetic ablation of the anti-inflammatory cytokine IL-10.
- Published in:
- Human Molecular Genetics, 2014, v. 23, n. 15, p. 3990, doi. 10.1093/hmg/ddu113
- By:
- Publication type:
- Article
Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice.
- Published in:
- Human Molecular Genetics, 2013, v. 22, n. 24, p. 4914, doi. 10.1093/hmg/ddt341
- By:
- Publication type:
- Article
Impaired viability of muscle precursor cells in muscular dystrophy with glycosylation defects and amelioration of its severe phenotype by limited gene expression.
- Published in:
- Human Molecular Genetics, 2013, v. 22, n. 15, p. 3003, doi. 10.1093/hmg/ddt157
- By:
- Publication type:
- Article
Matrix metalloproteinase-2 ablation in dystrophin-deficient mdx muscles reduces angiogenesis resulting in impaired growth of regenerated muscle fibers.
- Published in:
- Human Molecular Genetics, 2011, v. 20, n. 9, p. 1787, doi. 10.1093/hmg/ddr062
- By:
- Publication type:
- Article
Residual laminin-binding activity and enhanced dystroglycan glycosylation by LARGE in novel model mice to dystroglycanopathy.
- Published in:
- Human Molecular Genetics, 2009, v. 18, n. 4, p. 621, doi. 10.1093/hmg/ddn387
- By:
- Publication type:
- Article
ɛ-Sarcoglycan compensates for lack of α-sarcoglycan in a mouse model of limb-girdle muscular dystrophy.
- Published in:
- Human Molecular Genetics, 2005, v. 14, n. 6, p. 775, doi. 10.1093/hmg/ddi072
- By:
- Publication type:
- Article
Laminin α1 chain reduces muscular dystrophy in laminin α2 chain deficient mice.
- Published in:
- Human Molecular Genetics, 2004, v. 13, n. 16, p. 1775, doi. 10.1093/hmg/ddh190
- By:
- Publication type:
- Article
Erratum: G-CSF supports long-term muscle regeneration in mouse models of muscular dystrophy.
- Published in:
- Nature Communications, 2015, v. 6, n. 6, p. 7295, doi. 10.1038/ncomms8295
- By:
- Publication type:
- Article
A muscle-liver-fat signalling axis is essential for central control of adaptive adipose remodelling.
- Published in:
- Nature Communications, 2015, v. 6, n. 4, p. 6693, doi. 10.1038/ncomms7693
- By:
- Publication type:
- Article
G-CSF supports long-term muscle regeneration in mouse models of muscular dystrophy.
- Published in:
- Nature Communications, 2015, v. 6, n. 4, p. 6745, doi. 10.1038/ncomms7745
- By:
- Publication type:
- Article
Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy.
- Published in:
- Science Translational Medicine, 2018, v. 10, n. 437, p. 1, doi. 10.1126/scitranslmed.aan0713
- By:
- Publication type:
- Article
Induction of Pluripotent Stem Cells from a Manifesting Carrier of Duchenne Muscular Dystrophy and Characterization of Their X-Inactivation Status.
- Published in:
- Stem Cells International, 2017, p. 1, doi. 10.1155/2017/7906843
- By:
- Publication type:
- Article
C9orf72 and RAB7L1 regulate vesicle trafficking in amyotrophic lateral sclerosis and frontotemporal dementia.
- Published in:
- 2017
- By:
- Publication type:
- journal article
Deficiency of Cbl-b Gene Enhances Infiltration and Activation of Macrophages in Adipose Tissue and Causes Peripheral Insulin Resistance in Mice.
- Published in:
- Diabetes, 2007, v. 56, n. 10, p. 2511, doi. 10.2337/db06-1768
- By:
- Publication type:
- Article
Establishment of a Triple Quadrupole HPLC-MS Quantitation Method for Dystrophin Protein in Mouse and Human Skeletal Muscle.
- Published in:
- International Journal of Molecular Sciences, 2024, v. 25, n. 1, p. 303, doi. 10.3390/ijms25010303
- By:
- Publication type:
- Article
Periostin Is Required for the Maintenance of Muscle Fibers during Muscle Regeneration.
- Published in:
- International Journal of Molecular Sciences, 2021, v. 22, n. 7, p. 3627, doi. 10.3390/ijms22073627
- By:
- Publication type:
- Article
ATP-Induced Increase in Intracellular Calcium Levels and Subsequent Activation of mTOR as Regulators of Skeletal Muscle Hypertrophy.
- Published in:
- International Journal of Molecular Sciences, 2018, v. 19, n. 9, p. 2804, doi. 10.3390/ijms19092804
- By:
- Publication type:
- Article
Endogenous Multiple Exon Skipping and Back-Splicing at the DMD Mutation Hotspot.
- Published in:
- International Journal of Molecular Sciences, 2016, v. 17, n. 10, p. 1722, doi. 10.3390/ijms17101722
- By:
- Publication type:
- Article
The nSMase2/Smpd3 gene modulates the severity of muscular dystrophy and the emotional stress response in mdx mice.
- Published in:
- 2020
- By:
- Publication type:
- journal article
Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy.
- Published in:
- Nature, 2011, v. 478, n. 7367, p. 127, doi. 10.1038/nature10456
- By:
- Publication type:
- Article
Early phase 2 trial of TAS‐205 in patients with Duchenne muscular dystrophy.
- Published in:
- Annals of Clinical & Translational Neurology, 2020, v. 7, n. 2, p. 181, doi. 10.1002/acn3.50978
- By:
- Publication type:
- Article
A phase I study of TAS‐205 in patients with Duchenne muscular dystrophy.
- Published in:
- Annals of Clinical & Translational Neurology, 2018, v. 5, n. 11, p. 1338, doi. 10.1002/acn3.651
- By:
- Publication type:
- Article
Molecular analysis of the duchenne muscular dystrophy gene in patients with becker muscular dystrophy presenting with dilated cardiomyopathy.
- Published in:
- Muscle & Nerve, 1993, v. 16, n. 11, p. 1161, doi. 10.1002/mus.880161104
- By:
- Publication type:
- Article
Vascular abnormalities in the placenta of Chst14<sup>−/−</sup> fetuses: implications in the pathophysiology of perinatal lethality of the murine model and vascular lesions in human CHST14/D4ST1 deficiency.
- Published in:
- Glycobiology, 2018, v. 28, n. 2, p. 80, doi. 10.1093/glycob/cwx099
- By:
- Publication type:
- Article
Structural and biochemical characterization of O-mannose-linked human natural killer-1 glycan expressed on phosphacan in developing mouse brains.
- Published in:
- Glycobiology, 2014, v. 24, n. 3, p. 314, doi. 10.1093/glycob/cwt116
- By:
- Publication type:
- Article
Protein-anchoring Strategy for Delivering Acetylcholinesterase to the Neuromuscular Junction.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 7, p. 1384, doi. 10.1038/mt.2012.34
- By:
- Publication type:
- Article
Long-term Engraftment of Multipotent Mesenchymal Stromal Cells That Differentiate to Form Myogenic Cells in Dogs With Duchenne Muscular Dystrophy.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 1, p. 168, doi. 10.1038/mt.2011.181
- By:
- Publication type:
- Article
Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 5, p. 830, doi. 10.1038/mt.2011.59
- By:
- Publication type:
- Article
The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 1, p. 9, doi. 10.1038/mt.2010.219
- By:
- Publication type:
- Article