Funded Research Projects

CHAR0312: Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
Principal Investigator: Wagner, Kathryn
The purpose of this research is to collect blood and skin samples from Duchenne Muscular Dystrophy (DMD) patients ages 4 years and above with specific genetic mutations within the dystrophin gene...
Myostatin regulates fate of satellite cells in dystrophic muscle
Principal Investigator: Wagner, Kathryn
The goal of this proposal is to determine whether myostatin, expressed in both skeletal muscle and fibroblasts, regulates the conversion of satellite cells to fibroblasts and whether this conversion...
Partnering to treat an Orphan Disease Duchenne Muscular Dystrophy
Principal Investigator: Wagner, Kathryn
The goal of this program is to develop an investigational drug by Sanofi for a new indication, Duchenne Muscular Dystrophy.  The program includes preclinical toxicology and efficacy studies as...
A Three Dimensional Environment for Skeletal Muscle Stem Cell Transplantation
Principal Investigator: Wagner, Kathryn
The purpose of this collaborative project is to develop and test and injectable scaffold embedded with muscle stem cells and a Pfizer drug to facilitate muscle regeneration. Cell based therapies...
Biomarkers for Therapy of FSHD
Principal Investigator: Wagner, Kathryn
The goal of this Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center is to define modifying genes of FSHD and to determine, through novel animal models of FSHD, whether these are...
Acyl-CoA Synthetase ACSVL3 in Malignant Glioma: Metabolism and Oncogenic Cellular
Principal Investigator: Watkins, Paul
Malignant glioma accounts for a significant percentage of brain tumors. As these tumors are typically refractory to treatment, there is a need for new and novel therapeutic approaches. Levels of...
Understanding Mechanisms of Lower Extremity Strengthening in Women Heterozygous for X-ALD
Principal Investigator: Zackowski, Kathleen
Sponsored by the ELA Foundation. X-linked adrenoleukodystrophy (X-ALD), a sex-linked progressive neurodegenerative disease, is caused by a defect in the ABCD1 gene. The disease is expressed in...
Predicting Intervention Responsiveness to Improve Rehabilitation in People with Multiple Sclerosis
Principal Investigator: Zackowski, Kathleen
Sponsored by the National Multiple Sclerosis Society -- RG 4668-A-1. Multiple sclerosis (MS) is an inflammatory disease of the central nervous system (CNS) distinguished pathologically by...