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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\u2019s Disease, Huntington\u2019s 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.<\/jats:p>","DOI":"10.1007\/s00415-025-12940-5","type":"journal-article","created":{"date-parts":[[2025,2,22]],"date-time":"2025-02-22T11:30:44Z","timestamp":1740223844000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Genetic therapies for movement disorders\u00a0-\u00a0current status"],"prefix":"10.1007","volume":"272","author":[{"given":"J.","family":"Sartorelli","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J.","family":"Ng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A. 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JN has held Sponsored Research Agreements with AskBio Europe, Rocket Pharma, Bloomsbury Genetic Therapies and Consultancy contracts with Albion Venture Capital, Helex Bio. JN holds equity in Bloomsbury Genetic Therapies. AAR is a co-founder and has equity in Bloomsbury Genetic Therapies Ltd. Scientific advisory board and has equity in Nuntius Therapeutics. AAR holds patents on \u201cCompositions and methods for treating Parkinson\u2019s Disease (USSN 63\/437,437)\u201d and \u201cGene therapy of Niemann-Pick disease type c (WO2021181096A1)\u201d. SNW Scientific and Manufacturing Advisory Board Member, Forge Biologics and Co-founder of, and equity in, Bloomsbury Genetic Therapies. SNW holds patents on \u201cCompositions and methods for treating Parkinson\u2019s Disease (USSN 63\/437,437)\u201d, \u201cGene Therapy for Dopamine Transporter Deficiency Syndrome (PCT\/GB2101958.3)\u201d and \u201cGene Therapy Vector for eEf1A2 and uses thereof (US application no. 63055775; PCT\/EP2020\/071028)\u201d. MAK is a cofounder and consultant to Bloomsbury Genetic Therapies, and have received honoraria and consultancy fees from PTC Therapeutics.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}}],"article-number":"220"}}