The Administrative Core A will be responsible for the planning, development, coordination, and overall administration of the University of Massachusetts Medical School (UMMS) Wellstone MD CRC focused on Facioscapulohumeral Muscular Dystrophy (FSHD). Center leadership will continue under Dr. Charles Emerson, now at UMMS, and Dr. Louis Kunkel at Children's Boston, both highly experienced research administrators and accomplished scientists. The administrative unit for Core A will be located at UMMS, which has substantial resources, personnel and experience for management of a U54 Center. Core A will be the administrative hub to manage all Center resources and communications and carry out policies and procedures that establish and energized environment to enable effective communication and collaboration among all the Center constituents, including investigators, trainees, FSHD patients and advocacy groups, industry, NIH and the public. The Center will promote its mission through its website, which disseminates information on Center activities and research, and through its partnership with patient advocacy groups, particularly the FSH Society, which co-sponsors FSHD Research Workshops and Researcher-Patient Networking meetings to engage patients, researchers, trainees and NIH in our Center mission. Through Core A, the Center will promote the distribution of muscle cell lines from its Wellstone FSHD Cell Repository as a unique FSHD bio-resource. Internal committees will enable Center leadership, investigators and trainees to effectively utilize resources, organize internal dataflow and communications, and receive feedback on Center management, research progress and training. Regularly-scheduled Webex meetings will focus on data sharing and research planning, and an annual Wellstone Center Retreat at UMMS will bring investigators and trainees face-to-face with a Center Advisory Committee (CAC) and NIH program leaders to review research progress, set goals and align Center activities with the NIH Wellstone Center Network.
The Administrative Core A will provide administrative and communications support to enable the UMMS Wellstone Center to achieve its research mission to understand the underlying causes of Facioscapulohumeral Muscular Dystophy (FSHD), to develop therapeutics to treat this prevalent and debilitating disease, and to train research and clinical leaders in FSHD and muscular dystrophy research.
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|Wallace, Lindsay M; Saad, Nizar Y; Pyne, Nettie K et al. (2018) Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Mol Ther Methods Clin Dev 8:121-130|
|Giesige, Carlee R; Wallace, Lindsay M; Heller, Kristin N et al. (2018) AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD. JCI Insight 3:|
|Chagarlamudi, Hema; Corbett, Alastair; Stoll, Marion et al. (2017) Bone health in facioscapulohumeral muscular dystrophy: A cross-sectional study. Muscle Nerve 56:1108-1113|
|Eichinger, Katy; Heatwole, Chad; Heininger, Susanne et al. (2017) Validity of the 6 minute walk test in facioscapulohumeral muscular dystrophy. Muscle Nerve 55:333-337|
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|Shaw, Natalie D; Brand, Harrison; Kupchinsky, Zachary A et al. (2017) SMCHD1 mutations associated with a rare muscular dystrophy can also cause isolated arhinia and Bosma arhinia microphthalmia syndrome. Nat Genet 49:238-248|
|Widrick, Jeffrey J; Alexander, Matthew S; Sanchez, Benjamin et al. (2016) Muscle dysfunction in a zebrafish model of Duchenne muscular dystrophy. Physiol Genomics 48:850-860|
|Chen, Jennifer Cj; King, Oliver D; Zhang, Yuanfan et al. (2016) Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics. Mol Ther 24:1405-11|
|Ansseau, Eugénie; Eidahl, Jocelyn O; Lancelot, Céline et al. (2016) Homologous Transcription Factors DUX4 and DUX4c Associate with Cytoplasmic Proteins during Muscle Differentiation. PLoS One 11:e0146893|
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