Martínez de Lagrán Cabredo, MaríaElizalde-Torrent, AleixParedes, RogerClotet, BonaventuraDierssen, Mara2022-11-022022-11-022022Martinez de Lagran M, Elizalde-Torrent A, Paredes R, Clotet B, Dierssen M. Lamivudine, a reverse transcriptase inhibitor, rescues cognitive deficits in a mouse model of down syndrome. J Cell Mol Med. 2022 Aug;26(15):4210-5. DOI: 10.1111/jcmm.174111582-1838http://hdl.handle.net/10230/54648An elevated activity of retrotransposons is increasingly recognized to be implicated in a wide range of neurodegenerative and neurodevelopmental diseases. Down syndrome (DS) is the most common genetic disorder associated with intellectual disability and a genetic form of Alzheimer's disease. For this reason, we hypothesized that treatment with reverse transcriptase inhibitors could ameliorate DS phenotypes. In this proof of concept study, we treated trisomic (Ts65Dn) mice, a model of DS, with lamivudine, a reverse transcriptase inhibitor. We detected a significant improvement of neurobehavioural phenotypes, and a complete rescue of the hippocampal-dependent recognition memory upon treatment with lamivudine. Despite clinical studies in patients with DS are warranted, this study lays the groundwork for a novel and actionable therapeutic approach.application/pdfeng© 2022 The Authors. Journal of Cellular and Molecular Medicine published by Foundation for Cellular and Molecular Medicine and John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.info:eu-repo/semantics/articlehttp://dx.doi.org/10.1111/jcmm.17411Down syndromeLamivudineMemoryRetrotransposonsReverse transcriptase inhibitorinfo:eu-repo/semantics/openAccess