The Administrative Core A will be responsible for the overall administration of the Wellstone MDRC. It will provide resources, personnel, governance, oversight and policies and procedures to promote aU54 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 externalMD CRC components and activities by providing financial, administrative and information technologymanagement and support for Cores and Projects. It facilitates the organization and scheduling of allWellstone 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. Itcoordinates Wellstone MD CRC communications through meetings with key scientific participants on aninternal Local Executive Committee (LEG); with advisors ad patient representatives on an external CenterAdvisory Committee (CAC), with NIH staff and will help develop intra-Wellstone MD CRC, trans-WellstoneMD CRC, trans-NIH research activities, resources for the NIH MDCC Action Plan, and for the externalcommunity 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 properallocation of funds for each core and project member. Core A will also be responsible for coordinatingreviews 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 andbiomaterials among researchers, clinicians and trainees by displaying raw materials and data, finished dataand publications. It will help facilatate the evaluation and transfer of materials to the appropriate internal andpublic 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 scientificcommunities through education and training programs, and patient networking meetings, annual researchsymposium(s), and annual research training retreats in order to accelerate research breakthroughs onFSHD.

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 #
1U54HD060848-01
Application #
7536204
Study Section
Special Emphasis Panel (ZAR1-KM-J (M1))
Project Start
2008-09-10
Project End
2013-08-31
Budget Start
2008-09-10
Budget End
2009-08-31
Support Year
1
Fiscal Year
2008
Total Cost
$77,864
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
Sakellariou, Paraskevi; O'Neill, Andrea; Mueller, Amber L et al. (2016) Neuromuscular electrical stimulation promotes development in mice of mature human muscle from immortalized human myoblasts. Skelet Muscle 6:4
Choudhury, Sourav R; Fitzpatrick, Zachary; Harris, Anne F et al. (2016) In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy. Mol Ther 24:1247-57
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

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