Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppression

• DOI: 10.1038/srep12166
• Publication Date: 2015-07-15T10:43:45Z
• Authors: Amber L. Southwell; Stephen E.P. Smith; Tessa R. Davis; Nicholas S. Caron; Erika B. Villanueva; Yuanyun Xie; Jennifer A. Collins; Min Li Ye; Aaron Sturrock; Blair R. Leavitt; Adam G. Schrum; Michael R. Hayden
• Keywords: Huntingtin Protein

Abstract Quantitation of huntingtin protein in the brain is needed, both as a marker Huntington disease (HD) progression and for use clinical gene silencing trials. Measurement cerebrospinal fluid could be biomarker huntingtin, but traditional quantitation methods have failed to detect fluid. Using micro-bead based immunoprecipitation flow cytometry (IP-FCM), we developed highly sensitive mutant detection assay. The sensitivity IP-FCM enables accurate HD patients model mice, demonstrating that levels reflect levels, increasing with stage decreasing following suppression. This technique has potential applications research tool biomarker.