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Non-invasive tracking of disease progression in young dystrophic muscles using multi-parametric MRI at 14T.
- Published in:
- PLoS ONE, 2018, v. 13, n. 10, p. 1, doi. 10.1371/journal.pone.0206323
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- Publication type:
- Article
Recombinant adeno-associated viral ( rAAV) vectors mediate efficient gene transduction in cultured neonatal and adult microglia.
- Published in:
- Journal of Neurochemistry, 2016, v. 136, p. 49, doi. 10.1111/jnc.13081
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- Publication type:
- Article
Stem-cell biology: A move in the right direction.
- Published in:
- Nature, 2006, v. 444, n. 7119, p. 552, doi. 10.1038/nature05406
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- Publication type:
- Article
Removing the Immune Response From Muscular Dystrophy Research.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 10, p. 1821, doi. 10.1038/mt.2013.209
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- Publication type:
- Article
Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting.
- Published in:
- Human Molecular Genetics, 2014, v. 23, n. 25, p. 6697, doi. 10.1093/hmg/ddu388
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- Publication type:
- Article
Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting.
- Published in:
- Human Molecular Genetics, 2012, v. 21, n. 25, p. 1, doi. 10.1093/hmg/ddu388
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- Publication type:
- Article
Expression of the dystrophin isoform Dp116 preserves functional muscle mass and extends lifespan without preventing dystrophy in severely dystrophic mice.
- Published in:
- Human Molecular Genetics, 2011, v. 20, n. 24, p. 4978, doi. 10.1093/hmg/ddr433
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- Publication type:
- Article
Molecular and cellular adaptations to chronic myotendinous strain injury in mdx mice expressing a truncated dystrophin.
- Published in:
- Human Molecular Genetics, 2008, v. 17, n. 24, p. 3975, doi. 10.1093/hmg/ddn301
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- Publication type:
- Article
Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy.
- Published in:
- Human Molecular Genetics, 2008, v. 17, n. 16, p. 2507, doi. 10.1093/hmg/ddn151
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- Publication type:
- Article
Functional capacity of dystrophins carrying deletions in the N-terminal actin-binding domain.
- Published in:
- Human Molecular Genetics, 2007, v. 16, n. 17, p. 2105, doi. 10.1093/hmg/ddm158
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- Publication type:
- Article
A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy.
- Published in:
- Human Molecular Genetics, 2006, v. 15, n. 10, p. 1610, doi. 10.1093/hmg/ddl082
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- Publication type:
- Article
Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.
- Published in:
- Nature Communications, 2017, v. 8, n. 6, p. 16007, doi. 10.1038/ncomms16007
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- Publication type:
- Article
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.
- Published in:
- Nature Communications, 2017, v. 8, n. 2, p. 14454, doi. 10.1038/ncomms14454
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- Publication type:
- Article
An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells.
- Published in:
- Nature Communications, 2013, v. 4, n. 3, p. 1549, doi. 10.1038/ncomms2550
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- Publication type:
- Article
Sarcolemma-localized nNOS is required to maintain activity after mild exercise.
- Published in:
- 2008
- By:
- Publication type:
- Letter
DIAPHRAGM MUSCLE STRIP PREPARATION FOR EVALUATION OF GENE THERAPIES IN mdx MICE.
- Published in:
- Clinical & Experimental Pharmacology & Physiology, 2008, v. 35, n. 7, p. 725, doi. 10.1111/j.1440-1681.2007.04865.x
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- Publication type:
- Article
The mdx-amplification-resistant mutation system assay, a simple and rapid polymerase chain reaction-based detection of the mdx allele.
- Published in:
- 1996
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- Publication type:
- journal article
Muscle Structure Influences Utrophin Expression in <i>mdx</i> Mice.
- Published in:
- PLoS Genetics, 2014, v. 10, n. 6, p. 1, doi. 10.1371/journal.pgen.1004431
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- Publication type:
- Article
The Polyproline Site in Hinge 2 Influences the Functional Capacity of Truncated Dystrophins.
- Published in:
- PLoS Genetics, 2010, v. 6, n. 5, p. 1, doi. 10.1371/journal.pgen.1000958
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- Publication type:
- Article
Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 8, p. 1501, doi. 10.1038/mt.2012.111
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- Publication type:
- Article
Age-dependent Dystrophin Loss and Genetic Reconstitution Establish a Molecular Link Between Dystrophin and Heart Performance During Aging.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 10, p. 1821, doi. 10.1038/mt.2011.120
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- Publication type:
- Article
Gene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 1, p. 36, doi. 10.1038/mt.2010.205
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- Publication type:
- Article
Immune Responses to AAV in Canine Muscle Monitored by Cellular Assays and Noninvasive Imaging.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 3, p. 617, doi. 10.1038/mt.2009.294
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- Publication type:
- Article
Dystrophin Delivery to Muscles of mdx Mice Using Lentiviral Vectors Leads to Myogenic Progenitor Targeting and Stable Gene Expression.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 1, p. 206, doi. 10.1038/mt.2009.253
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- Publication type:
- Article
Evaluation of Vascular Delivery Methodologies to Enhance rAAV6-mediated Gene Transfer to Canine Striated Musculature.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 8, p. 1427, doi. 10.1038/mt.2009.116
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- Publication type:
- Article
Erratum to “Efficient Transduction of Skeletal Muscle Using Vectors Based on Adeno-associated Virus Serotype 6”.
- Published in:
- 2009
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- Publication type:
- Correction notice
Microutrophin Delivery Through rAAV6 Increases Lifespan and Improves Muscle Function in Dystrophic Dystrophin/Utrophin-deficient Mice.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 9, p. 1539, doi. 10.1038/mt.2008.149
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- Publication type:
- Article
Recombinant Adeno-associated Virus Transduction and Integration.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 7, p. 1189, doi. 10.1038/mt.2008.103
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- Publication type:
- Article
Emergent Dilated Cardiomyopathy Caused by Targeted Repair of Dystrophic Skeletal Muscle.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 5, p. 832, doi. 10.1038/mt.2008.52
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- Publication type:
- Article
Systemic Microdystrophin Gene Delivery Improves Skeletal Muscle Structure and Function in Old Dystrophic mdx Mice.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 4, p. 657, doi. 10.1038/mt.2008.28
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- Publication type:
- Article
Systemic Administration of Micro-dystrophin Restores Cardiac Geometry and Prevents Dobutamine-induced Cardiac Pump Failure.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 6, p. 1086, doi. 10.1038/sj.mt.6300144
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- Publication type:
- Article
Sustained AAV-mediated Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy with a Brief Course of Immunosuppression.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 6, p. 1160, doi. 10.1038/sj.mt.6300161
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- Publication type:
- Article
Design of Tissue-specific Regulatory Cassettes for High-level rAAV-mediated Expression in Skeletal and Cardiac Muscle.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 2, p. 320, doi. 10.1038/sj.mt.6300027
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- Publication type:
- Article
35. Systemic Administration of rAAV6-Microdystrophin Preserves Muscle Function and Extends Lifespan in the Dystrophin-/Utrophin- Mouse Model of Severe Muscular Dystrophy.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S15, doi. 10.1016/j.ymthe.2006.08.048
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- Publication type:
- Article
561. Novel Tissue-Specific Regulatory Cassettes Direct High-Level Transgene Expression in Skeletal and Cardiac Muscle.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S216, doi. 10.1016/j.ymthe.2006.08.634
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- Publication type:
- Article
Gene Therapy Strategies for Duchenne Muscular Dystrophy Utilizing Recombinant Adeno-associated Virus Vectors.
- Published in:
- Molecular Therapy, 2006, v. 13, n. 2, p. 241, doi. 10.1016/j.ymthe.2005.11.001
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- Publication type:
- Article
Phenotypic Improvement of Dystrophic Muscles by rAAV/Microdystrophin Vectors Is Augmented by Igf1 Codelivery
- Published in:
- Molecular Therapy, 2005, v. 12, n. 3, p. 441, doi. 10.1016/j.ymthe.2005.04.001
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- Publication type:
- Article
1127. Perivascular CD45<sup>-</sup>:Sca-1<sup>+</sup>:CD34<sup>-</sup> Cells Are Derived from Bone Marrow Cells and Participate in Dystrophic Skeletal Muscle Regeneration
- Published in:
- 2005
- By:
- Publication type:
- Abstract
1040. Donor Origin of Multipotent Adult Progenitor Cells in Radiation Chimeras
- Published in:
- 2005
- By:
- Publication type:
- Abstract
793. Efficient Expression of the 6 kb Mini-Dystrophin Gene by Trans-Splicing Adeno-Associated Viral (AAV) Vector Restores the Entire Dystrophin-Associated Glycoprotein Complex and Reduces Contraction-Induced Damage in the Mdx Mouse Model of Duchenne Muscular Dystrophy
- Published in:
- 2005
- By:
- Publication type:
- Abstract
791. Immunity to AAV-Mediated Gene Therapy in a Random-Bred Canine Model of Duchenne Muscular Dystrophy
- Published in:
- 2005
- By:
- Publication type:
- Abstract
513. Novel Characteristics of Heparin Binding by Adeno-Associated Virus Serotype 6
- Published in:
- 2005
- By:
- Publication type:
- Abstract
266. Development of rAAV-Based Skeletal and Cardiac Muscle Regulatory Cassettes for Gene Therapy of Duchenne Muscular Dystrophy
- Published in:
- 2005
- By:
- Publication type:
- Abstract
Adeno-Associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury
- Published in:
- Molecular Therapy, 2005, v. 11, n. 2, p. 245, doi. 10.1016/j.ymthe.2004.09.013
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- Publication type:
- Article
Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6
- Published in:
- Molecular Therapy, 2004, v. 10, n. 4, p. 671, doi. 10.1016/j.ymthe.2004.07.016
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- Publication type:
- Article
Sustained Human Factor VIII Expression in Hemophilia A Mice Following Systemic Delivery of a Gutless Adenoviral Vector
- Published in:
- Molecular Therapy, 2002, v. 5, n. 1, p. 63, doi. 10.1006/mthe.2001.0510
- By:
- Publication type:
- Article
Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review.
- Published in:
- BioDrugs, 2024, v. 38, n. 1, p. 95, doi. 10.1007/s40259-023-00632-3
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- Publication type:
- Article
Cas9 immunity creates challenges for CRISPR gene editing therapies.
- Published in:
- Nature Communications, 2018, v. 9, n. 1, p. 1, doi. 10.1038/s41467-018-05843-9
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- Publication type:
- Article
Biomarkers for Duchenne muscular dystrophy progression: impact of age in the mdx tongue spared muscle.
- Published in:
- Skeletal Muscle, 2023, v. 13, n. 1, p. 1, doi. 10.1186/s13395-023-00325-z
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- Publication type:
- Article
Biomarkers for Duchenne muscular dystrophy progression: impact of age in the mdx tongue spared muscle.
- Published in:
- Skeletal Muscle, 2023, v. 13, n. 1, p. 1, doi. 10.1186/s13395-023-00325-z
- By:
- Publication type:
- Article