Genetic therapies for movement disorders ‑ current status
Date
2025-02
Authors
Journal Title
Journal ISSN
Volume Title
Publisher
Springer
Abstract
Movement 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.
Description
Keywords
Genetic therapy, Movement disorder, Gene, Viral vector, Brain
Citation
Sartorelli, 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/s00415-025-12940-5