Carvalho, SofiaSantos, Juliana InêsMoreira, LucianaDuarte, Ana JoanaGaspar, PauloRocha, HugoEncarnação, MarisaRibeiro, DiogoBarbosa Almeida, MatildeGonçalves, MarianaDavid, HugoMatos, LilianaAmaral, OlgaDiogo, LuísaFerreira, SaraSantos, ConstançaMartins, EsmeraldaPrata, Maria JoãoPereira de Almeida, LuísAlves, SandraCoutinho, Maria Francisca2025-03-122025-03-122024-03-21Int J Mol Sci. 2024 Mar 21;25(6):3546. doi: 10.3390/ijms250635461661-6596http://hdl.handle.net/10400.18/10435This article belongs to the Special Issue The Lysosome in Human Health and Diseases.Among the many lysosomal storage disorders (LSDs) that would benefit from the establishment of novel cell models, either patient-derived or genetically engineered, is mucopolysaccharidosis type II (MPS II). Here, we present our results on the establishment and characterization of two MPS II patient-derived stem cell line(s) from deciduous baby teeth. To the best of our knowledge, this is the first time a stem cell population has been isolated from LSD patient samples obtained from the dental pulp. Taking into account our results on the molecular and biochemical characterization of those cells and the fact that they exhibit visible and measurable disease phenotypes, we consider these cells may qualify as a valuable disease model, which may be useful for both pathophysiological assessments and in vitro screenings. Ultimately, we believe that patient-derived dental pulp stem cells (DPSCs), particularly those isolated from human exfoliated deciduous teeth (SHEDs), may represent a feasible alternative to induced pluripotent stem cells (iPSCs) in many labs with standard cell culture conditions and limited (human and economic) resources.engLysosomal Storage DiseasesDental Pulp Stem Cells (DPSCs)Disease ModelingIn vitro ModelsInduced Pluripotent Stem Cells (iPSCs)Mucopolysaccharidosis Type IIStem Cells From Human Exfoliated Deciduous Teeth (SHEDs)Doenças Lisossomais de SobrecargaDoenças GenéticasGenética Humanaresearch article10.3390/ijms2506354638542525