Pharmacodynamic biomarkers responsive to mutant huntingtin lowering in a Huntington's disease mouse model

This is the authors' abstract. We don't add key points for this paper.

• Candidate PET imaging biomarkers Pde10a, mHTT and D 1 R are responsive to early m Htt lowering. • Candidate CSF biomarkers Snc4b and Ace are responsive to early m Htt lowering. • Early intervention with moderate (∼50 %) whole-body m Htt lowering is required to delay progression of phenotypes; late intervention resulted in minimal benefits.