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NIH to Grant $3M for Muscular Dystrophy Centers

NEW YORK (GenomeWeb News) – The National Institutes of Health will grant $3 million in 2010 to fund three muscular dystrophy research centers that will conduct cooperative studies to address development of therapeutics for muscular dystrophy and will include genomics and biomarker research.

Under the Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers grant program, the NIH will fund researchers who will join a network of existing centers to foster translation of new scientific findings and technological developments for use in muscular dystrophy treatments.

These centers may contain a mixture of basic, translational, or clinical research, as long as it is directed to the steps required for therapeutic development, including development of diagnostics and biomarkers to characterize or stratify patient populations, in vitro assay development, candidate therapeutic efficacy screening, and other efforts.

In studies focused on disease mechanisms, researchers may pursue identification of genes and characterization of biochemicals involved in muscular dystrophy, and they may identify and validate downstream pathogenic mechanisms for therapeutic development.

These studies also may focus on development and validation of gene tests or minimally invasive diagnostic techniques for muscular dystrophy that may allow identification and stratification of cohorts for clinical studies and trials.

Researchers also may develop diagnostic tools and biomarkers that could be used as measures in clinical trials. In particular, these could include tools and biomarkers that may provide short-term surrogates for the prediction of the long-term impact of a candidate therapeutic.

Research programs focused on therapeutics may engage in a wide variety of approaches to the types of muscular dystrophy, including determining optimal gene constructs for gene therapy, and development of agents for gene modification or repair and methods to test their delivery.

The program is led by the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Arthritis and Muscoloskeletal and Skin Diseases, the National Hearth, Lung, and Blood Institute, and the National Institute of Neurological Disorders and Stroke.

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