Effects of mutant huntingtin inactivation on Huntington disease-related behaviours in the BACHD mouse model.

ABSTRACT:

Aims

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.

Methods

We generated mutant BACHD mice with either a widespread or circuit-specific inactivation of mutant HTT by using Cre recombinase (Cre) under the nestin promoter or the adenosine A_2A receptor promoter respectively.

SUBMITTER: Cheong RY 

PROVIDER: S-EPMC8247873 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature