The pace of scientific advancements, development of new clinical guidelines, and "instant access" to web based data and information is rapidly changing and often requires flexible guidance and oversight. Such oversight is crucial as experimental therapies may outpace the resources available to translate these findings into clinical trials, especially in myotonic dystrophy type 1 (DM1). The main goals of the Administrative Core of our Wellstone Center are to coordinate and oversee seamless communication of all activities in the renewal application of our Wellstone Center.
The specific aims of the Administrative Core are to coordinate and integrate the components &activities as well as the utilization of funds by the 2 scientific projects, the Scientific Resources Core and the Training &Educational Core that comprise our Wellstone Center. The Administrative Core will play an especially important role in coordinating the interactions between the newly established Myotonic Dystrophy Clinical Research Network (DM CRN) that consists of 5 sites (Ohio State University, Stanford University, University of Florida, University of Kansas, and University of Rochester). We have proposed collaborative studies within the DM CRN to validate endpoints, biomarkers, and patient reported outcomes as described in Project 1. Our Administrative Core gains vitality and effectiveness from prior collaborations with each of these sites and locally, from the experience of our staff and investigators, who have coordinated activities over the past two cycles of our Wellstone Center (2003-2013). The necessary communication network, knowledge of storage and confidentiality requirements, data tracking software, and computer facilities to operate a large-scale, multi-center, NIH funded, project are already in place. We will enhance our communications with various committees, both internally and externally, to facilitate smooth operations of our Wellstone activities and interactions with the DM CRN. In addition, the Administrative Core will bring together the robust assets that are available within the Medical Center, such as, the recently funded Clinical &Translational Sciences Institute and our Departmental resources, to facilitate the activities of our scientific projects and cores.
The monitoring and communication responsibilities of our Administrative Core are vital to implement the activities proposed in our new Wellstone Center in order to prepare for promising experimental therapies in myotonic dystrophy type 1. Our administrate goals are synergized by collaborations within and outside the Wellstone Network, resources of our Medical Center, and long-standing interactions with patients.
|Hoskins, Jason W; Ofori, Leslie O; Chen, Catherine Z et al. (2014) Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects. Nucleic Acids Res 42:6591-602|
|Thornton, Charles A (2014) Myotonic dystrophy. Neurol Clin 32:705-19, viii|
|Statland, Jeffrey M; Tawil, Rabi (2014) Risk of functional impairment in Facioscapulohumeral muscular dystrophy. Muscle Nerve 49:520-7|
|Batra, Ranjan; Charizanis, Konstantinos; Manchanda, Mini et al. (2014) Loss of MBNL leads to disruption of developmentally regulated alternative polyadenylation in RNA-mediated disease. Mol Cell 56:311-22|
|Heatwole, Chad; Bode, Rita; Johnson, Nicholas et al. (2014) Myotonic Dystrophy Health Index: initial evaluation of a disease-specific outcome measure. Muscle Nerve 49:906-14|
|Rzuczek, Suzanne G; Gao, Yu; Tang, Zhen-Zhi et al. (2013) Features of modularly assembled compounds that impart bioactivity against an RNA target. ACS Chem Biol 8:2312-21|
|Hilbert, James E; Ashizawa, Tetsuo; Day, John W et al. (2013) Diagnostic odyssey of patients with myotonic dystrophy. J Neurol 260:2497-504|
|Childs-Disney, Jessica L; Stepniak-Konieczna, Ewa; Tran, Tuan et al. (2013) Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules. Nat Commun 4:2044|
|Kalman, Lisa; Tarleton, Jack; Hitch, Monica et al. (2013) Development of a genomic DNA reference material panel for myotonic dystrophy type 1 (DM1) genetic testing. J Mol Diagn 15:518-25|
|Coonrod, Leslie A; Nakamori, Masayuki; Wang, Wenli et al. (2013) Reducing levels of toxic RNA with small molecules. ACS Chem Biol 8:2528-37|
Showing the most recent 10 out of 56 publications