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They are typically progressive, very disabling and frequently involve the nervous system. Currently available symptomatic therapies provide modest benefit at best. The development of interventions that interfere with the natural history of these diseases is a priority. A common pathogenic process shared by most TRDs is the presence of toxicity from the messenger RNA or protein encoded by the gene harboring the abnormal expansion. Strategies to interfere with the expression of these genes using different molecular approaches are being pursued and have reached the clinical stage. This review will summarize the significant progress made in this field in the last few years, focusing on three main areas: the discovery of biomarkers of disease progression and target engagement, advances in preclinical studies for the polyglutamine ataxias and the initial clinical application in myotonic dystrophy type 1 and Huntington\u2019s disease.<\/jats:p>","DOI":"10.1093\/hmg\/ddz138","type":"journal-article","created":{"date-parts":[[2019,6,11]],"date-time":"2019-06-11T19:11:19Z","timestamp":1560280279000},"page":"R80-R87","source":"Crossref","is-referenced-by-count":25,"title":["Recent advances in molecular therapies for neurological disease: triplet repeat disorders"],"prefix":"10.1093","volume":"28","author":[{"given":"Pedro","family":"Gonzalez-Alegre","sequence":"first","affiliation":[{"name":"Department of Neurology, the University of Pennsylvania, Philadelphia, PA 19104, USA"},{"name":"Raymond G. Perelman Center for Cellular and Molecular Therapy, The Children\u2019s Hospital of Philadelphia, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2019,6,22]]},"reference":[{"key":"2019101710390129100_ref1","doi-asserted-by":"crossref","first-page":"R13","DOI":"10.1186\/gb-2003-4-2-r13","article-title":"Genome-wide analysis of microsatellite repeats in humans: their abundance and density in specific genomic regions","volume":"4","author":"Subramanian","year":"2003","journal-title":"Genome Biol."},{"key":"2019101710390129100_ref2","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1038\/ng.3461","article-title":"Abundant contribution of short tandem repeats to gene expression variation in humans","volume":"48","author":"Gymrek","year":"2016","journal-title":"Nat. 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