[HTML][HTML] TGF-β–driven muscle degeneration and failed regeneration underlie disease onset in a DMD mouse model

…, MW Hogarth, M Nearing, P Adusumalli, CB Tully… - JCI insight, 2020 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy (DMD) is a chronic muscle disease characterized by poor
myogenesis and replacement of muscle by extracellular matrix. Despite the shared genetic …

[PDF][PDF] TNF-α-induced microRNAs control dystrophin expression in Becker muscular dystrophy

AA Fiorillo, CR Heier, JS Novak, CB Tully, KJ Brown… - Cell reports, 2015 - cell.com
The amount and distribution of dystrophin protein in myofibers and muscle is highly variable
in Becker muscular dystrophy and in exon-skipping trials for Duchenne muscular dystrophy. …

Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy

CR Heier, Q Yu, AA Fiorillo, CB Tully… - Life science …, 2019 - life-science-alliance.org
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 …

Serum miRNAs are pharmacodynamic biomarkers associated with therapeutic response in pediatric inflammatory bowel disease

…, CR Heier, L Diaz-Calderon, CB Tully… - Inflammatory Bowel …, 2020 - academic.oup.com
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 …

[PDF][PDF] Muscle weakness in myositis: MicroRNA‐mediated dystrophin reduction in a myositis mouse model and human muscle biopsies

TB Kinder, CR Heier, CB Tully… - Arthritis & …, 2020 - Wiley Online Library
Objective Muscle inflammation is a feature in myositis and Duchenne muscular dystrophy (
DMD ). Autoimmune mechanisms are thought to contribute to muscle weakness in patients …

[HTML][HTML] Estrogen receptor, inflammatory, and FOXO transcription factors regulate expression of myasthenia gravis-associated circulating microRNAs

AA Fiorillo, CR Heier, YF Huang, CB Tully… - Frontiers in …, 2020 - frontiersin.org
MicroRNAs (miRNAs) are small non-coding RNA molecules that regulate important intracellular
biological processes. In myasthenia gravis (MG), a disease-specific pattern of elevated …

The X‐linked Becker muscular dystrophy (bmx) mouse models Becker muscular dystrophy via deletion of murine dystrophin exons 45–47

CR Heier, NM McCormack, CB Tully… - Journal of Cachexia …, 2023 - Wiley Online Library
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…

Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone

AA Fiorillo, CB Tully, JM Damsker… - Physiological …, 2018 - journals.physiology.org
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 …

[HTML][HTML] Multi-omics identifies circulating miRNA and protein biomarkers for facioscapulohumeral dystrophy

CR Heier, A Zhang, NY Nguyen, CB Tully… - Journal of Personalized …, 2020 - mdpi.com
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 …

[HTML][HTML] Interrogation of dystrophin and dystroglycan complex protein turnover after exon skipping therapy

…, AA Fiorillo, R Hindupur, CB Tully… - Journal of …, 2021 - content.iospress.com
Recently, the Food and Drug Administration granted accelerated approvals for four exon
skipping therapies–Eteplirsen, Golodirsen, Viltolarsen, and Casimersen–for Duchenne …