Research and Development Unit, Department of Human Genetics, National Institute of Health Doutor Ricardo Jorge, INSA I.P., Rua Alexandre Herculano, 321, 4000-055 Porto, Portugal.
Center for the Study of Animal Science-Institute of Sciences, Technologies and Agro-Environment, CECA-ICETA, University of Porto, Praça Gomes Teixeira, Apartado 55142, 4051-401 Porto, Portugal.
Int J Mol Sci. 2024 Mar 21;25(6):3546. doi: 10.3390/ijms25063546.
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.
在许多可能受益于新型细胞模型建立的溶酶体贮积症（LSD）中，黏多糖贮积症 II 型（MPS II）就是其中之一。在这里，我们介绍了从乳牙牙髓中分离出两种 MPS II 患者来源的干细胞系的结果。据我们所知，这是首次从 LSD 患者样本中分离出干细胞群体，这些样本来自牙髓。考虑到我们对这些细胞的分子和生化特征的研究结果，以及它们表现出明显和可测量的疾病表型，我们认为这些细胞可能符合有价值的疾病模型的标准，这对于生理病理评估和体外筛选都可能是有用的。最终，我们相信患者来源的牙髓干细胞（DPSCs），特别是那些从人脱落乳牙（SHED）中分离出来的细胞，在许多具有标准细胞培养条件和有限（人力和经济）资源的实验室中，可能是诱导多能干细胞（iPSCs）的可行替代物。
