Current Projects: 2008-2013
The design of our current URMC Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC) builds on the synergy of our robust Medical Center environment and on the opportunity to identify more effective treatments for myotonic dystrophy type 1 (DM1). Our Wellstone Center researchers have a long-standing interest in the pathomechanisms responsible for DM1, and the members of this team have already worked together in previous collaborative projects. Most have actively participated in our previously funded Wellstone Center. A brief description of our two current projects is listed below.
Project 1: “Pathogenesis and Progression in Myotonic Dystrophy”
The Aims of this project are to evaluate bench to bedside aspects of disease progression. The goals of project are to study the genetic instability of DM1, to study the relationship between various biochemical markers of DM1 and muscle impairment, and to conduct a longitudinal study of a large cohort of DM1 patients to help determine which outcome measures are most sensitive to monitor disease progression and suitable as endpoints in clinical trials.
Project 2: “Experimental Therapy of Myotonic Dystrophy”
The Aims of this project encompass gene therapy, oligonucleotide therapy, and small molecule (drug) therapy for myotonic dystrophy.
Last updated 5/18/2009