Christopher B. Tully
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View article: Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping
Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping Open
Duchenne muscular dystrophy (DMD) is a progressive muscle disease caused by the absence of dystrophin protein. One current DMD therapeutic strategy, exon skipping, produces a truncated dystrophin isoform using phosphorodiamidate morpholino…
View article: The glucocorticoid receptor acts locally to protect dystrophic muscle and heart during disease
The glucocorticoid receptor acts locally to protect dystrophic muscle and heart during disease Open
Absence of dystrophin results in muscular weakness, chronic inflammation and cardiomyopathy in Duchenne muscular dystrophy (DMD). Pharmacological corticosteroids are the DMD standard of care; however, they have harsh side effects and uncle…
View article: Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice
Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice Open
View article: Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping
Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping Open
Duchenne muscular dystrophy (DMD) is a progressive muscle disease caused by the absence of dystrophin protein. One current DMD therapeutic strategy, exon skipping, produces a truncated dystrophin isoform using phosphorodiamidate morpholino…
View article: The X‐linked Becker muscular dystrophy (<i>bmx</i>) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45–47
The X‐linked Becker muscular dystrophy (<i>bmx</i>) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45–47 Open
Background Becker muscular dystrophy (BMD) is a genetic neuromuscular disease of growing importance caused by in‐frame, partial loss‐of‐function mutations in the dystrophin ( DMD ) gene. BMD presents with reduced severity compared with Duc…
View article: Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy
Interrogation of Dystrophin and Dystroglycan Complex Protein Turnover After Exon Skipping Therapy Open
Recently, the Food and Drug Administration granted accelerated approvals for four exon skipping therapies –Eteplirsen, Golodirsen, Viltolarsen, and Casimersen –for Duchenne Muscular Dystrophy (DMD). However, these treatments have only demo…
View article: Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy
Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy Open
The development of therapeutics for muscle diseases such as facioscapulohumeral dystrophy (FSHD) is impeded by a lack of objective, minimally invasive biomarkers. Here we identify circulating miRNAs and proteins that are dysregulated in ea…
View article: Serum miRNAs Are Pharmacodynamic Biomarkers Associated With Therapeutic Response in Pediatric Inflammatory Bowel Disease
Serum miRNAs Are Pharmacodynamic Biomarkers Associated With Therapeutic Response in Pediatric Inflammatory Bowel Disease Open
Background We sought to identify microRNAs (miRNAs) associated with response to anti-TNF-α or glucocorticoids in children with inflammatory bowel disease (IBD) to generate candidate pharmacodynamic and monitoring biomarkers. Methods Clinic…
View article: TGF-β–driven muscle degeneration and failed regeneration underlie disease onset in a DMD mouse model
TGF-β–driven muscle degeneration and failed regeneration underlie disease onset in a DMD mouse model Open
Duchenne muscular dystrophy (DMD) is a chronic muscle disease characterized by poor myogenesis and replacement of muscle by extracellular matrix. Despite the shared genetic basis, severity of these deficits varies among patients. One sourc…
View article: Estrogen Receptor, Inflammatory, and FOXO Transcription Factors Regulate Expression of Myasthenia Gravis-Associated Circulating microRNAs
Estrogen Receptor, Inflammatory, and FOXO Transcription Factors Regulate Expression of Myasthenia Gravis-Associated Circulating microRNAs Open
MicroRNAs (miRNAs) are small non-coding RNA molecules that regulate important intracellular biological processes. In myasthenia gravis (MG), a disease-specific pattern of elevated circulating miRNAs has been found, and proposed as potentia…
View article: Muscle Weakness in Myositis: MicroRNA‐Mediated Dystrophin Reduction in a Myositis Mouse Model and Human Muscle Biopsies
Muscle Weakness in Myositis: MicroRNA‐Mediated Dystrophin Reduction in a Myositis Mouse Model and Human Muscle Biopsies Open
Objective Muscle inflammation is a feature in myositis and Duchenne muscular dystrophy ( DMD ). Autoimmune mechanisms are thought to contribute to muscle weakness in patients with myositis. However, a lack of correlation between the extent…
View article: Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy
Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy Open
Cardiomyopathy is a leading cause of death for Duchenne muscular dystrophy. Here, we find that the mineralocorticoid receptor (MR) and glucocorticoid receptor (GR) can share common ligands but play distinct roles in dystrophic heart and sk…
View article: Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone Open
Corticosteroids are highly prescribed and effective anti-inflammatory drugs but the burden of side effects with chronic use significantly detracts from patient quality of life, particularly in children. Developing safer steroids amenable t…
View article: TNF-α-Induced microRNAs Control Dystrophin Expression in Becker Muscular Dystrophy
TNF-α-Induced microRNAs Control Dystrophin Expression in Becker Muscular Dystrophy Open