Modelling Lysosomal Storage Disorders in an innovative way: Establishment and Characterization of Stem Cell Lines from Human Exfoliated Deciduous Teeth of Mucopolysaccharidoses Type II Patients

Carvalho, S.; Santos, J.I.; Moreira, L.; Duarte, A.J.; Gaspar, P.; Rocha, H.; Encarnação, M.; Ribeiro, D.; Barbosa Almeida, M.; Gonçalves, M.; David, H.; Matos, L.; Amaral, O.; Diogo, L.; Ferreira, S.; Santos, C.; Martins, E.; Prata, M.J.; Pereira de Almeida, L.; Alves, S.; Coutinho, M.F. Modeling Lysosomal Storage Disorders in an Innovative Way: Establishment and Characterization of Stem Cell Lines from Human Exfoliated Deciduous Teeth of Mucopolysaccharidosis Type II Patients. Int. J. Mol. Sci. 2024, 25, 3546. Carvalho, S.; Santos, J.I.; Moreira, L.; Duarte, A.J.; Gaspar, P.; Rocha, H.; Encarnação, M.; Ribeiro, D.; Barbosa Almeida, M.; Gonçalves, M.; David, H.; Matos, L.; Amaral, O.; Diogo, L.; Ferreira, S.; Santos, C.; Martins, E.; Prata, M.J.; Pereira de Almeida, L.; Alves, S.; Coutinho, M.F. Modeling Lysosomal Storage Disorders in an Innovative Way: Establishment and Characterization of Stem Cell Lines from Human Exfoliated Deciduous Teeth of Mucopolysaccharidosis Type II Patients. Int. J. Mol. Sci. 2024, 25, 3546.

Abstract

Among the many Lysosomal Storage Disorders (LSDs) that would benefit from the establish-ment of novel cell models, either patient-derived or genetically engineered, is Mucopolysaccha-ridosis type II (MPS II). In fact, even though a specific therapeutic approach does exist for this disorder (Enzyme Replacement Therapy, ERT, with recombinant human IDS) and haematopoiet-ic stem cell transplantation (HSCT) may also hold promise whenever the disorder is diagnosed soon enough, both approaches have their own drawbacks. Here we present our results on the establishment and characterization of two MPS II pa-tient-derived stem cell line(s) from deciduous baby teeth. To the best of our knowledge, this is the first time a stem cell population is isolated from LSD patient samples obtained from the oral cavity. Taking into account our results on the molecular and biochemical characterization of those cells and our first insights on their subcellular MPS phenotype, we consider these cells will be a valuable tool for both pathophysiological assessments and drugs screenings. Ultimately, we believe patient-derived dental pulp stem cells (DPSC) may represent a feasible alternative to induced pluripotent stem cells (iPSC) in many labs with standard cell culture con-ditions and limited (human and economic) resources.

Keywords

Mucopolysaccharidosis type II; Disease Modelling; in vitro models; induced Pluripotent Stem Cells (iPSCs); Dental Pulp Stem Cells (DPSCs); SHED: Stem cells from Human Exfoliated Deciduous teeth (SHEDs)

Subject

Biology and Life Sciences, Biology and Biotechnology

Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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