The National Institute of Neurological Disorders and Stroke (NINDS) and the Office of Rare Disorders (ORD) invite grant applications for central nervous system (CNS) therapy development for lysosomal storage disorders.
Lysosomal storage disorders include about 50 metabolic diseases that collectively affect approximately 1 in 5000 live births. Each of these diseases has heterogeneous pathophysiology and clinical manifestations resulting from deficient activity of specific hydrolases. In some cases, the genetic defect can be in an activator protein for a lysosomal hydrolase or a transporter protein for the metabolites. All of these deficiencies lead to a characteristic pathological accumulation and storage of the substrate for that enzyme in the lysosomes. The consequent accumulation of undigested metabolites in lysosomes leads to multi-systemic dysfunction, including progressive neurologic deterioration, mental retardation, organomegaly, blindness, and early death.
For more information, potential applicants should contact Dr. Danilo Tagle, Program Director, Neurogenetics Cluster, NINDS; telephone: 301-496-5745; e-mail: email@example.com. For a more detailed description of this announcement, visit http://grants.nih.gov/grants/guide/pa-files/PAS-07-195.html.