Past Event! Note: this event has already taken place.
2021-22 Health Sciences Seminar Series – Dr. Utkarsh Dang
October 14, 2021 at 12:00 PM to 1:00 PM
| Location: | This event will be held online via Zoom. Please contact healthsciences@carleton.ca for Zoom details. |
The Department of Health Sciences is pleased to announce the next seminar in our seminar series.
Presenter: Dr. Utkarsh Dang, Assistant Professor, Department of Health Sciences, Carleton University
Title: Serum Biomarkers and Clinical Severity in Young Steroid-Naïve Duchenne Muscular Dystrophy Boys
Duchenne muscular dystrophy (DMD) is caused by loss of dystrophin in muscle, and while all patients share the primary gene and biochemical defect, there is considerable patient–patient variability in clinical symptoms. We developed multivariate models of serum protein biomarkers that explained observed variation, using functional outcome measures as proxies for severity. Serum samples from 39 steroid-naïve DMD boys 4 to <7 years enrolled into a clinical trial of vamorolone (NCT02760264) were studied. Weighted correlation network analysis was used
for unsupervised clustering of 1305 proteins quantified using SOMAscan® aptamer profiling to define highly representative and connected proteins. Multivariate models of biomarkers were obtained for time to stand performance (strength phenotype; 17 proteins) and 6 min walk performance (endurance phenotype; 17 proteins) including some shared proteins. Identified proteins were further tested with associations of mRNA expression with histological severity of muscle from dystrophinopathy patients (n = 28) and normal controls (n = 6). We show that
performance of DMD boys was effectively modeled with serum proteins, proximal strength associated with growth and remodeling pathways and muscle endurance centered on TGFβ and fibrosis pathways in muscle.