The Administrative Core A will be responsible for the overall administration of the Wellstone MD RC. It will provide resources, personnel, governance, oversight and policies and procedures to promote a U54 center environment through enhanced communication to facilitate collaboration among investigators, Datient communities, NIH and the public. It will organize, direct and integrate the flow of internal and external MD CRC components and activities by providing financial, administrative and information technology management and support for Cores and Projects. It facilitates the organization and scheduling of all Wellstone MD CRC-related meetings and events across a distributed geographical center using internet, websites, video- and tele-conferencing, Wiki collaboration software and physical meeting space. It coordinates Wellstone MD CRC communications through meetings with key scientific participants on an internal Local Executive Committee (LEG);with advisors ad patient representatives on an external Center Advisory Committee (CAC), with NIH staff and will help develop intra-Wellstone MD CRC, trans-Wellstone MD CRC, trans-NIH research activities, resources for the NIH MDCC Action Plan, and for the external community of scientists to facilate developing areas of facioscapulohumeral muscular dystrophy (FSHD) research. It will plan, review, maintain all financial and personnel records, and assure the the proper allocation of funds for each core and project member. Core A will also be responsible for coordinating reviews and evaluation of pre and post-doctoral trainees with the Education and Training Core. The Administrative Core will communicate and promote the use of the Cell Core C resources and biomaterials among researchers, clinicians and trainees by displaying raw materials and data, finished data and publications. It will help facilatate the evaluation and transfer of materials to the appropriate internal and public repositories for tissues, cells and mice to muscular dystrophy and FSHD research endeavors. The Administrative Core achieves greater value through outreach to academic and industrial scientific communities through education and training programs, and patient networking meetings, annual research symposium(s), and annual research training retreats in order to accelerate research breakthroughs on FSHD.

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Specialized Center--Cooperative Agreements (U54)
Project #
5U54HD060848-02
Application #
7917476
Study Section
Special Emphasis Panel (ZAR1)
Project Start
Project End
Budget Start
2009-09-01
Budget End
2010-08-31
Support Year
2
Fiscal Year
2009
Total Cost
$79,176
Indirect Cost
Name
Boston Biomedical Research Institute
Department
Type
DUNS #
058893371
City
Watertown
State
MA
Country
United States
Zip Code
02472
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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
Ansseau, Eugénie; Vanderplanck, Céline; Wauters, Armelle et al. (2017) Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy (FSHD). Genes (Basel) 8:
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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