LAUSR

Huntington disease (HD) is a fatal neurodegenerative disorder with no disease‐modifying treatments approved so far. Ongoing clinical trials are attempting to reduce huntingtin (HTT) expression in the central nervous system (CNS) using different strategies. Yet, the distribution and timing of HTT‐lowering therapies required for a beneficial clinical effect is less clear. Here, we investigated whether HD‐related behaviours could be prevented by inactivating mutant HTT at different disease stages and to varying degrees in an experimental model.