Coutinho, Maria FranciscaSantos, Juliana InêsMatos, LilianaAlves, Sandra2021-03-132021-03-132020-11http://hdl.handle.net/10400.18/7461Here we present an alternative approach to generate patient-derived MPS III neuronal cell lines through a non-invasive, cost effective approach using dental pulp stem cells (DPSC). The plasticity of DPSCs has been demonstrated by several independent teams, who have shown that they self-maintain through several passages, holding potential to give rise to a variety of cells including functionally active neurons. Nevertheless, to the best of our knowledge, this sort of technology hasn’t yet been applied to samples obtained from LSD patients. Our goal is to establish one such protocol in house, to generate MPS III neurons from normally shed deciduous “baby” teeth. Once established, those neuronal cell lines will constitute a valuable platform to address further address RNA drugs’ efficiency in vitro.engLysosomal Storage DiseasesMPS IIITherapeutic RNA DrugsDental Pulp Stem CellsPatient-derived MPS III Neuronal Cell LinesDoenças GenéticasTerapias de RNAconference object