Duke University

James R Burke

Warren J Strittmatter

Jeffery M Vance

Jan J Enghild

Yoshitaka Nagai

Compounds that selectively bind to expanded polyglutamine repeats are disclosed. Such compounds are characterized in that they bind to a first polyglutamine peptide consisting of 60 glutamine residues under conditions in which they do not bind to a second polyglutamine peptide consisting of 20 glutamine residues. Conjugates of such compounds, nucleic acids encoding the same, and methods of use thereof are also disclosed.

Compounds that selectively bind to expanded polyglutamine repeat domains and methods of use thereof

GAPDH binds to expanded polyglutamine regions which are found in proteins encoded by genes with CAG repeat domains, which genes cause inherited neurodegenerative disorders and are associated with certain psychiatric disorders. Therapeutic methods are based on inhibiting the binding of GAPDH to expanded polyglutamine regions. A method of screening compounds for the ability to inhibit binding of GAPDH to polyglutamine regions is described.

Growing community of inventors

Chapel Hill, NC, United States of America

James R Burke