Waddington, S. N.2025-03-252025-02Sartorelli, J., Ng, J., Rahim, A.A. et al. Genetic therapies for movement disorders - current status. J Neurol 272, 220 (2025). https://doi.org/10.1007/s00415-025-12940-50340-5354 (print)1432-1459 (online)10.1007/s00415-025-12940-5https://hdl.handle.net/10539/44437Movement disorders are a group of heterogeneous neurological conditions associated with alterations of tone, posture and voluntary movement. They may either occur in isolation or as part of a multisystemic condition. More recently, the advent of next generation sequencing technologies has facilitated better understanding of the underlying causative genes and molecular pathways, thereby identifying targets for genetic therapy. In this review, we summarize the advances in genetic therapy approaches for both hyperkinetic and hypokinetic movement disorders, including Parkinson’s Disease, Huntington’s Disease and rarer monogenic conditions of childhood onset. While there have been significant advances in the field, multiple challenges remain, related to safety, toxicity, efficacy and brain biodistribution, which will need to be addressed by the next generation of genetic therapies.en© The Author(s) 2025. Open Access, This article is licensed under a Creative Commons Attribution 4.0 International License.Genetic therapyMovement disorderGeneViral vectorBrainArticleSDG-3: Good health and well-being