The Administrative Core (J. Mendell, PI) will provide scientific and fiscal oversight of and direction to the MDCRC Program. The Core will build upon the existing expertise of the Neuromuscular Clinical Research Program at the Research Institute at Nationwide Children's Hospital (NCH). The translational research staff already assembled by the PI, Dr. Mendell, has a demonstrated expertise in designing and implementing cutting-edge clinical research projects. The staff's experience in coordinating large multi-center collaborative programs is directly relevant to administration of a Weilstone MDCRC, and includes experience in coordination of communication among multiple sites;quality control and document tracking;data management;data mining and reporting;milestone and task management;and fiscal accountability. Access to patients will be facilitated by the United Dystrophinopathy Project, a large NIH-funded multicenter genotype/phenotype and natural history consortium directed by Dr. Kevin Flanigan, who has recently joined the NCH Center for Gene Therapy. Dr. Mendell has extensive experience in the design and performance of DMD clinical trials, including unique experience in the performance of gene transfer trials, and both he and Dr. Flanigan have served as consultants for the design of trials of gene transfer or gene correction (exon skipping and nonsense suppression) trials that are currently underway. The Core will be responsible for establishing and maintaining effective communications and cooperation among investigators both inside the NCH MDCRC and among the wider Weilstone network. In this role, it will develop clear internal and external procedures for monitoring and evaluating the research projects and core facilities;establish mechanisms for managing and sharing data, animal models, and other resources among investigators;and provide administrative support for the development of both extramural funded and industry-sponsored translational research projects, and for the Educational Core activities of the MDCRC. The Administrative Core will also establish a Center Advisory Committee, and develop the framework to ensure the contribution of this Committee to the oversight of the Center's research and translational activities.
The Administrative Core is critical to the function of the entire MDCRC. The well-established expertise of the staff assembled by Dr. Mendell will facilitate the functions of the MDCRC as outlined in the overall project, and provide detailed and timely oversight to Investigators within the MDCRC, and collaborators throughout the Weilstone network.
|Mendell, Jerry R; Sahenk, Zarife; Al-Zaidy, Samiah et al. (2017) Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes. Mol Ther 25:870-879|
|Eidahl, Jocelyn O; Giesige, Carlee R; Domire, Jacqueline S et al. (2016) Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4. Hum Mol Genet 25:4577-4589|
|Mendell, Jerry R; Sahenk, Zarife; Malik, Vinod et al. (2015) A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy. Mol Ther 23:192-201|
|Sondergaard, Patricia C; Griffin, Danielle A; Pozsgai, Eric R et al. (2015) AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models. Ann Clin Transl Neurol 2:256-70|
|Heller, Kristin N; Montgomery, Chrystal L; Shontz, Kimberly M et al. (2015) Human ?7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient Mice. Hum Gene Ther 26:647-56|
|Al-Zaidy, Samiah A; Sahenk, Zarife; Rodino-Klapac, Louise R et al. (2015) Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy. J Neuromuscul Dis 2:185-192|
|Yalvac, Mehmet E; Arnold, William David; Hussain, Syed-Rehan A et al. (2014) VIP-expressing dendritic cells protect against spontaneous autoimmune peripheral polyneuropathy. Mol Ther 22:1353-1363|
|Chicoine, L G; Montgomery, C L; Bremer, W G et al. (2014) Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. Mol Ther 22:338-347|
|Chicoine, Louis G; Rodino-Klapac, Louise R; Shao, Guohong et al. (2014) Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin ?2 surrogates. Mol Ther 22:713-24|
|Mendell, Jerry R; Lloyd-Puryear, Michele (2013) Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy. Muscle Nerve 48:21-6|
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