Dystrophin
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Subject Areas on Research
- A beta-spectrin isoform from Drosophila (beta H) is similar in size to vertebrate dystrophin.
- AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.
- AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.
- An ankyrin-based mechanism for functional organization of dystrophin and dystroglycan.
- Ankyrin-B interactions with spectrin and dynactin-4 are required for dystrophin-based protection of skeletal muscle from exercise injury.
- Assembly of the dystrophin-associated protein complex does not require the dystrophin COOH-terminal domain.
- Ataluren treatment of patients with nonsense mutation dystrophinopathy.
- Biochemical and biomechanical characteristics of dystrophin-deficient mdx3cv mouse lens.
- Breathing in Duchenne muscular dystrophy: translation to therapy.
- Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.
- Clinical and genetic characterization of manifesting carriers of DMD mutations.
- Cloning and characterization of cytokeratins 8 and 19 in adult rat striated muscle. Interaction with the dystrophin glycoprotein complex.
- Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.
- Duchenne muscular dystrophy and steroids: pharmacologic treatment in the absence of effective gene therapy.
- Dystrophin and utrophin isoforms are expressed in glia, but not neurons, of the avian parasympathetic ciliary ganglion.
- Dystrophin immunity in Duchenne's muscular dystrophy.
- Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.
- Electrical stimulation increases hypertrophy and metabolic flux in tissue-engineered human skeletal muscle.
- Essential roles of the dystrophin-glycoprotein complex in different cardiac pathologies.
- Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice.
- Expression of the 71 kDa dystrophin isoform (Dp71) evaluated by gene targeting.
- Familial X-linked myalgia and cramps: a nonprogressive myopathy associated with a deletion in the dystrophin gene.
- Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
- GWAS Identifies New Loci for Painful Temporomandibular Disorder: Hispanic Community Health Study/Study of Latinos.
- Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.
- Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy.
- In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.
- Inefficient dystrophin expression after cord blood transplantation in Duchenne muscular dystrophy.
- Inherited deletion at Duchenne dystrophy locus in normal male.
- Interactions between dystrophin and the sarcolemma membrane.
- Intragenic duplication and divergence in the spectrin superfamily of proteins.
- Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
- Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
- Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy.
- Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies.
- Motor axonopathies in a mouse model of Duchenne muscular dystrophy.
- Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.
- Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators.
- Novel Rod Domain Duplication in Dystrophin Resulting in X-Linked Dilated Cardiomyopathy.
- Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.
- Prenatal diagnosis of Duchenne muscular dystrophy by fetal muscle biopsy.
- Progress in therapy for Duchenne muscular dystrophy.
- Progress towards gene therapy for Duchenne muscular dystrophy.
- Pseudometabolic presentation of dystrophinopathy due to a missense mutation.
- Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.
- STAT3 Regulates Self-Renewal of Adult Muscle Satellite Cells during Injury-Induced Muscle Regeneration.
- Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial.
- Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin.
- Serum biomarkers associated with baseline clinical severity in young steroid-naïve Duchenne muscular dystrophy boys.
- Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy.
- Soluble miniagrin enhances contractile function of engineered skeletal muscle.
- Stimulation of calcineurin signaling attenuates the dystrophic pathology in mdx mice.
- Suppression of revertant fibers in mdx mice by expression of a functional dystrophin.
- Systemic production of human granulocyte colony-stimulating factor in nonhuman primates by transplantation of genetically modified myoblasts.
- Tenascin-C expression in dystrophin-related muscular dystrophy.
- The CINRG Becker Natural History Study: Baseline characteristics.
- Vascular amyloid alters astrocytic water and potassium channels in mouse models and humans with Alzheimer's disease.