Novel Thio-Ether Analogues as a Treatment for Huntington's Disease
Lead Inventor: Juan Marugan (NCATS)
Inventors: Ehud Goldin (NHGRI), Omid Motabar (NHGRI), Samarjit Patnaik (NCATS), Ellen Sidransky (NHGRI), Noel Southall (NCATS), Wendy Westbroek (NHGRI), Wei Zheng (NCATS)
Ref. No.: E-258-2010/0
Abstract: This technology is a collection of small molecules screened for their ability to prevent or reduce the cytotoxic effects of the protein, Huntingtin. Huntington's disease is a neurodegenerative disorder due to a dominantly acting expansion of a CAG trinucleotide repeat in exon 1 of the Huntington (HTT) gene resulting in production of the altered (mutant) protein Huntingtin, which has a long chain of polyglutamine (poly Q) attached to the exon 1 encoded protein sequence.