NEW YORK (GenomeWeb News) – The National Institute for Neurological Disorders and Stroke will grant up to $4.5 million in 2011 to support research projects that will validate novel molecular targets for disease-modifying therapeutic intervention in Huntington's disease.
The goal of the program is to test hypotheses that could materially increase the evidence that modulating the biological activity of a certain molecular target could slow or even reverse the progression of the disease.
An inherited and fatal autosomal neurodegenerative disorder, Huntingdon's disease has a genetic basis which bears a CAG repeat sequence that is carried by around 120,000 Americans.
Targets for the research will have a demonstrated causal relationship with HD when it is manipulated genetically or pharmacological in vitro or invertebrate models of HD, said NIH. Further validation of eligible targets will involve genetic or pharmacological manipulation of the target in rodent or other in vivo mammalian HD models.
These targets may include receptors, intracellular or intercellular signaling molecules, molecular chaperones, transcriptional regulators, enzymes, structural elements, electron carriers, ions, or small molecules.
The targets researchers use may have been identified in a number of ways, including biochemical analysis of protein-protein interactions with mutant or wild type Huntington protein; in silico pathway analysis; altered gene expression; altered intracellular distribution; linkage with biological mechanisms that are relevant to HD pathophysiology; and/or through genome-wide assessment of modifiers of HD pathophysiology in cell-based or invertebrate models of HD such as C. elegans or Drosophila.