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.

Public Health Relevance

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.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
5U54NS048843-12
Application #
8733762
Study Section
Special Emphasis Panel (ZNS1-SRB-S)
Project Start
Project End
Budget Start
2014-09-01
Budget End
2015-08-31
Support Year
12
Fiscal Year
2014
Total Cost
$104,749
Indirect Cost
$36,509
Name
University of Rochester
Department
Type
DUNS #
041294109
City
Rochester
State
NY
Country
United States
Zip Code
14627
Carrell, Samuel T; Tang, Zhenzhi; Mohr, Sabine et al. (2018) Detection of expanded RNA repeats using thermostable group II intron reverse transcriptase. Nucleic Acids Res 46:e1
Trembley, Michael A; Quijada, Pearl; Agullo-Pascual, Esperanza et al. (2018) Mechanosensitive Gene Regulation by Myocardin-Related Transcription Factors Is Required for Cardiomyocyte Integrity in Load-Induced Ventricular Hypertrophy. Circulation 138:1864-1878
Auerbach, David S; Biton, Yitschak; Polonsky, Bronislava et al. (2018) Risk of cardiac events in Long QT syndrome patients when taking antiseizure medications. Transl Res 191:81-92.e7
Sznajder, ?ukasz J; Thomas, James D; Carrell, Ellie M et al. (2018) Intron retention induced by microsatellite expansions as a disease biomarker. Proc Natl Acad Sci U S A 115:4234-4239
Wood, Libby; Bassez, Guillaume; Bleyenheuft, Corinne et al. (2018) Eight years after an international workshop on myotonic dystrophy patient registries: case study of a global collaboration for a rare disease. Orphanet J Rare Dis 13:155
Jauvin, Dominic; Chrétien, Jessina; Pandey, Sanjay K et al. (2017) Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice. Mol Ther Nucleic Acids 7:465-474
Skov, Martin; Dirksen, Robert T (2017) Trojan triplets: RNA-based pathomechanisms for muscle dysfunction in Huntington's disease. J Gen Physiol 149:49-53
Pinto, Belinda S; Saxena, Tanvi; Oliveira, Ruan et al. (2017) Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9. Mol Cell 68:479-490.e5
Thornton, Charles A; Wang, Eric; Carrell, Ellie M (2017) Myotonic dystrophy: approach to therapy. Curr Opin Genet Dev 44:135-140
Gadalla, S M; Hilbert, J E; Martens, W B et al. (2017) Pigmentation phenotype, photosensitivity and skin neoplasms in patients with myotonic dystrophy. Eur J Neurol 24:713-718

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