Veronika Bračič

Toward Biomarker-Guided Gene Therapy for Neurodegeneration

Fellow

Dept. of Drug Design and Pharmacology, University of Copenhagen

December 21, 2025

The project will explore how lowering the harmful mutant huntingtin protein in Huntington’s disease restores brain function, and whether these improvements can be detected in accessible biofluids such as cerebrospinal fluid and tears, reflecting changes inside specific brain cell types.

A key challenge in Huntington’s disease is the lack of reliable markers that detect early brain changes and monitor treatment effects. By identifying biofluid biomarkers that mirror brain and cell-specific responses, this project aims to improve how we evaluate and guide emerging therapies.

We will use gene therapy in a Huntington’s disease mouse model and collect brain tissue and biofluids. Using advanced mass spectrometry, we will map protein changes across biofluids,  brain regions, and specific cell types, linking molecular brain rescue to measurable biomarkers for future clinical trials.