Work package 1: Evaluate the efficiency of lentiviral constructs to silence mhtt in striatum and hypothalamus of rats

Not much is known about the (dys)function of mutant huntingtin in striatum and hypothalamus. The aim of work package 1 is to evaluate the efficiency of lentiviral constructs to silence mutant huntingtin in striatum and hypothalamus of rats.

We will take advantage of the transgene construct of our BACHD rat. Therefore, it is essential for this project that Cre recombinase can be successfully delivered into the targeted brain regions of the striatum and the hypothalamus. QPS and the University of Tuebingen are taking a new approach in this field by merging their know-how and resources.

• Firstly, the University of Tuebingen will transfer BACHD rats and knowledge on breeding, genotyping and phenotyping of BACHD rats to QPS
• Secondly, QPS will perform stereotactic injection of lentiviral vectors into targeted brain regions
• Thirdly, QPS and the University of Tuebingen will validate efficacy of lentiviral delivery of CRE recombinase in vitro and in vivo