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Publication : Lamivudine, a reverse transcriptase inhibitor, rescues cognitive deficits in a mouse model of down syndrome.

First Author  Martinez de Lagran M Year  2022
Journal  J Cell Mol Med Volume  26
Issue  15 Pages  4210-4215
PubMed ID  35762509 Mgi Jnum  J:327075
Mgi Id  MGI:7328029 Doi  10.1111/jcmm.17411
Citation  Martinez de Lagran M, et al. (2022) Lamivudine, a reverse transcriptase inhibitor, rescues cognitive deficits in a mouse model of down syndrome. J Cell Mol Med 26(15):4210-4215
abstractText  An 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.
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