The training of undergraduates, medical students, predoctoral and postdoctoral students, as well as the general public, organized by the Education and Training Core, is an integral part of this Wellstone Center proposal on """"""""Biomarkers for Therapy of FSHD."""""""" The core's goal is to train the next generation of laboratory and clinician scientists in muscular dystrophy. By targeting students at multiple different levels of training from medical students through postdoctoral fellows, the core aims to increase the number of investigators in the field and to provide them with skills, through mentoring and assistance, to facilitate their transistion to productive, independent careers in muscular dystrophy research. During the previous grant award period, the Education Core has been a particular strength of our Wellstone having initiated a undergraduate student course in muscular dystrophy, trained predoctoral and postdoctoral laboratory students and a clinician scientist, several of whom have gone on to receive independent awards. The Core will continue to provide funding for 2 trainees for all projects involved in this proposal and will continue to involve 12 students from our Wellstone's participating insitutions funded by other mechanisms per year. It will be directed by Drs. Wagner (Core Director), Emerson (PI and Core Co-Director) and Brown (Core Co-Director) who with oversight by the Center Advisory Committee and NIH staff, will work with Center investigators to recruit, appoint, fund and mentor trainees. To enhance training of the students and to educate more broadly all FSHD investigators as well as FSHD patients, the Educational Core will include an annual internal Wellstone retreat, a biennial meeting with a FSH patient group, and an international research conference on FSHD.

Public Health Relevance

FSHD research has gained considerable momentum in the past five years from the creation of this Wellstone as well as support of other collaborating groups. To maintain this momentum and to carry discoveries into the clinic, the number of both laboratory and clinical researchers trained in FSHD needs to grow. The Educational and Training Core of our Wellstone will address this need in the community.

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 #
2U54HD060848-07
Application #
8621157
Study Section
Special Emphasis Panel (ZNS1-SRB-S (57))
Project Start
Project End
Budget Start
2013-09-16
Budget End
2014-05-31
Support Year
7
Fiscal Year
2013
Total Cost
$159,272
Indirect Cost
$63,709
Name
University of Massachusetts Medical School Worcester
Department
Type
DUNS #
603847393
City
Worcester
State
MA
Country
United States
Zip Code
01655
Leung, Doris G; Wang, Xin; Barker, Peter B et al. (2018) Multivoxel proton magnetic resonance spectroscopy in facioscapulohumeral muscular dystrophy. Muscle Nerve 57:958-963
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

Showing the most recent 10 out of 34 publications