The National Institute of Neurological Disorders and Stroke (NINDS), National Institute of Diabetes and Digestive and Kidney
Diseases (NIDDK), National Institute of Mental Health (NIMH), National Cancer Institute (NCI), National Institute of Arthritis
and Musculoskeletal and Skin Diseases (NIAMS), and the Tuberous Sclerosis Alliance invite grant applications for research
aimed at understanding and treating tuberous sclerosis complex (TSC). *
TSC is a multi-system, genetic disease that causes benign tumors (hamartomas) to form at multiple sites throughout the body—primarily the brain, lungs, heart, kidney, skin, and eyes. The genes that cause TSC—TSC1 and TSC2—are known, an understanding of the pathways in which they act is increasing, and animal models that mimic certain features of the disease now exist. Thus, there is a remarkable opportunity to increase knowledge about the mechanisms that cause TSC, and to translate this knowledge into therapies for the disorder.
Potential areas of research interest include, but are not limited to: studies of the role of hamartin and tuberin in basic cellular processes and development; development of cell culture models of TSC and more sophisticated animal models for TSC; identification of the downstream targets of tuberin and hamartin, with particular emphasis on potential molecular targets for drug therapy; developmental, neuroanatomical, electrophysiological, and imaging studies intended to identify specific abnormalities in TSC patients or in animal models of TSC; elucidation of the molecular events that cause lesions to develop in specific tissues; investigation of the pathogenesis of TSC-associated skin lesions and testing of potential therapeutic compounds to treat these lesions; studies aimed at understanding and developing treatments for TSC-associated infantile spasms and epilepsies; assessment and treatment of TSC-associated cognitive and behavioral problems; investigation of the role of the TSC1 and TSC2 gene products in other disorders; pre-clinical screening of potential small-molecule or gene-based therapies in cellular or animal models of TSC; and neurodevelopmental and longitudinal studies of TSC patients that investigate the progression and inherent variability of the disease.
For more information, potential applicants should contact Dr. Robert Finkelstein, Associate Director for Extramural Research, NINDS, Neuroscience Center, 6001 Executive Boulevard, Room 3307, Bethesda, MD 20892; telephone: 301-496-9248; fax: 301-402-4370; e-mail: firstname.lastname@example.org.
*For a more detailed description of this program announcement, please visit the NIH web site at: http://grants.nih.gov/grants/guide/pa-files/PAS-05-085.html.