The National Institute of Neurological Disorders and Stroke (NINDS), the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), and the National Institute on Aging (NIA) encourage grant applications for research targeting diseases caused by protein misfolding or misprocessing.*
With recent advances in small molecule production, design, and screening it is now possible to create molecules which bind to mutant proteins with high specificity and stabilize a near native conformation that allows them to escape sorting by quality control systems into degradative pathways or mistargeting within the cell. Because so many mutant proteins—once folded and exported to their normal location—retain some functional activity, these “chemical chaperones” have the potential to rescue mutant phenotypes in a host of diseases.
Areas of research interest include, but are not limited to: development of robust and reliable primary and secondary assays that can be used to screen small molecules for efficacy in a model of an inherited disease due to protein misprocessing, mistargeting, misfolding, or aggregation; conducting low or high throughput screening of small molecule therapeutics; conducting low or high throughput screening of RNAi reagents; confirmation of the results from the screen via additional assays, characterization of the molecular mechanism, or other means; additional limited, medicinal chemistry, including the synthesis and testing of related compounds for enhanced efficacy; and preliminary animal model testing of efficacious compounds, including appropriate preliminary pharmacological studies and evaluation of off-target effects.
LETTERS OF INTENT RECEIPT DATE: August 15, 2005.
APPLICATION RECEIPT DATE: September 15, 2005.
For more information, potential applicants should contact Dr. Danilo Tagle, Program Director, Neurogenetics Cluster, NINDS, Neuroscience Center, 6001 Executive Boulevard, Room 2133, Bethesda, MD 20892; telephone: 301-496-5745; fax: 301-402-1501; e-mail: email@example.com.
*For a more detailed description of this program announcement, please visit the NIH web site at: http://grants.nih.gov/grants/guide/pa-files/PAR-05-056.html.