The four projects in this Program Project share the common theme of developing newer approaches to celland gene therapy, and using zebrafish models to accelerate improvements in understanding and treating themuscular dystrophies. The Program Pi'shave a long and extensive history of interaction and haveexchanged many ideas, biopsy samples, and other reagents over many years. This Program Project furtherstrengthens these collaborations to produce synergistic results that are beyond the scope of any onelaboratory. The services of the Clinical Specimen, Data Collection, and Expression Array Core (Core B) arean important means by which the Program Project will meet these goals. Core B allows for increasedefficiency and provides standardization to the analysis of data for each Project and the Program overall.Toward this end, the Aims of Core B are designed to maximize efficiency and minimize both administrativeand technical effort and expense.
Aim 1. To carry out all activities necessary for participant recruitment intothe Program Projects, including the following: a) identify and enroll participants for the Program Project; b)acquire comprehensive data and specimens from participants, including clinical information, blood samples,and muscle tissue samples; c) acquire control tissues and specimens; d) catalogue and track the clinical anddiagnostic data in a database; and e) maintain the HNDP web site (Projects served: 1, 2, 3, 4, Core C).
Aim2. To perform gene expression array analysis for the Program Project: a) Prepare zebrafish embryos andwhole fish, cells, and muscle samples for gene expression analysis, isolate mRNA, and synthesize labeledcDNA/cRNA for hybridization to the Affymetrix GeneChip array and/or the Illumina Sentrix BeadChip; b)Hybridize cDNA to the Affymetrix GeneChip array and/or the Illumina Sentrix BeadChip; and c) Transfergene expression data to ProjectManager, a HNDP database, for analysis in Core C. (Projects served: 1, 2, 3,Core C). Core B will interact extensively with Core C, where much of the analysis of the gene expressiondata will take place.

Agency
National Institute of Health (NIH)
Institute
National Institute of Neurological Disorders and Stroke (NINDS)
Type
Research Program Projects (P01)
Project #
2P01NS040828-06A1
Application #
7408433
Study Section
Special Emphasis Panel (ZNS1-SRB-E (24))
Project Start
Project End
Budget Start
2007-09-01
Budget End
2008-03-31
Support Year
6
Fiscal Year
2007
Total Cost
$163,379
Indirect Cost
Name
Children's Hospital Boston
Department
Type
DUNS #
076593722
City
Boston
State
MA
Country
United States
Zip Code
02115
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Salajegheh, Mohammad; Kong, Sek Won; Pinkus, Jack L et al. (2010) Interferon-stimulated gene 15 (ISG15) conjugates proteins in dermatomyositis muscle with perifascicular atrophy. Ann Neurol 67:53-63
Glover, Louise E; Newton, Kimberly; Krishnan, Gomathi et al. (2010) Dysferlin overexpression in skeletal muscle produces a progressive myopathy. Ann Neurol 67:384-93
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