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.

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.

Tobacco effects on ocular surface, meibomian glands, and corneal epithelium and the benefits of treatment with a lipid-based lubricant.

PURPOSE: Our aim was to evaluate the ocular surface in chronic smokers and to assess the benefit of sodium hyaluronate (SH) versus semi-fluorinated alkane (SFA) eyedrops on tear film, meibomian glands, and corneal epithelial thickness (CET). METHODS: This prospective randomized single-blinded study included smokers, who applied one eyedrop of Hyabak® on one eye (SH group) and one eyedrop of EvoTears® on the fellow eye (SFA group) 4 times daily for 2 months, and age-matched non-smokers. Ocular surface parameters, including tear film break-up time (TBUT) test and corneal fluorescein staining (CFS) score, lipid layer thickness (LLT), meibography (LipiView®), and CET measurements (Zeiss Cirrus HD-5000®), were assessed at baseline and after treatment. RESULTS: Seventy-eight eyes were included in the smokers group (39 in the SH subgroup and 39 in the SFA subgroup) and 42 eyes in the control group. At baseline, the smokers group had a higher prevalence of dry eye (100% vs 0%, p < 0.001) and of meibomian gland dysfunction (MGD) and lower CET measurements than controls (p < 0.05). TBUT, CFS, and LLT (controls vs SFA group: 64.02 ± 1.87 nm vs 49.56 ± 4.33 nm, p = 0.05) improved in the SFA subgroup after treatment, but not in the SH subgroup, and became equivalent to those of controls. Prevalence of dry eye decreased in the smokers group after treatment (controls vs SH group vs SFA group: 0% vs 12.82% vs 16.26%, p > 0.05). Meibomian gland morphological parameters and CET did not improve after treatment (p < 0.05). CONCLUSIONS: Smoking is associated with dry eye, MGD, and corneal epithelial thinning that seem to be only partially reversible with topical lubricants, preferably SFA.

Eficácia de um programa educacional baseado no empowerment na perceção de autoeficácia em utentes com diabetes / Effectiveness of an empowerment-based educational program in self-efficacy perception in patients with diabetes / Eficacia de un programa educativo basado en el empoderamiento en la percepción de la autoeficacia en pacientes con diabetes

Enquadramento: Estudos sugerem que modelos educativos baseados no empowerment, implementados em pessoas com diabetes, poderão fomentar a melhoria da autoeficácia percecionada. Esta variável é considerada relevante na autogestão do regime terapêutico. Objetivos: Avaliar a eficácia de um programa educacional baseado no empowerment, na perceção de autoeficácia, em utentes com diabetes mellitus tipo 2. Metodologia: Estudo quasi-experimental, com avaliação antes e após implementação do programa, com intervalo de 6 semanas. Amostra aleatória randomizada, constituída por 42 participantes (grupo experimental = 19; grupo de controlo = 23), inscritos numa unidade de saúde familiar da Administração Regional de Saúde do Centro. O Diabetes empowerment Scale - Short Form (DES-SF) foi o instrumento utilizado para determinar a eficácia do programa. Foram assegurados todos os procedimentos formais e éticos. Resultados: Os utentes submetidos a um programa educativo baseado no empowerment apresentaram uma maior perceção de autoeficácia (p = 0,000), comparativamente com aqueles que seguiram o regular protocolo de vigilância. Conclusão: Os programas educacionais baseados no empowerment parecem contribuir para uma melhoria da autoeficácia percebida, pelo que poderão ser assumidos como estratégias a aplicar a utentes com diabetes mellitus tipo 2.

Background: Studies suggest that empowerment-based educational models, implemented in patients with diabetes, may encourage the improvement of perceived self-efficacy. This variable is considered relevant in the therapeutic regimen self-management. Objectives: To evaluate the effectiveness of an empowerment-based educational program in patients with type 2 diabetes mellitus. Methodology: A quasi-experimental study, with two different evaluation moments, with a 6-week interval. A randomized sample of 42 participants (experimental group = 19; control group = 23), registered in a family health unit of the ARS of Central Portugal. The Diabetes empowerment Scale - Short Form (DES-SF) was the instrument chosen to determine the program's effectiveness. All formal and ethical procedures were assured. Results: Patients subjected to the empowerment-based educational program presented a higher self-efficacy perception (p = 0.000) than those who followed the regular surveillance protocol. Conclusion: empowerment-based educational programs seem to contribute to an improvement in perceived self-efficacy and should, therefore, be used as an educational strategy for patients with type 2 diabetes mellitus.

Marco contextual: Los estudios sugieren que los modelos educativos basados en el empoderamiento, implementados en personas con diabetes, pueden fomentar la mejora de la autoeficacia percibida. Esta variable se considera relevante en el autocontrol del régimen terapéutico. Objetivos: Evaluar la eficacia de un programa educativo basado en el empoderamiento y la percepción de la autoeficacia en pacientes con diabetes mellitus tipo 2. Metodología: Estudio cuasiexperimental, con evaluación antes y después de la implementación del programa, con un intervalo de 6 semanas. Muestra aleatoria, compuesta por 42 participantes (grupo experimental = 19; grupo de control = 23), inscritos en una unidad de salud familiar de la Administración Regional de Salud del Centro. La Diabetes empowerment Scale - Short Form (DES-SF) fue el instrumento utilizado para determinar la efectividad del programa. Se garantizaron todos los procedimientos formales y éticos. Resultados: Los pacientes que se sometieron a un programa educativo basado en el empoderamiento tuvieron una mayor percepción de la autoeficacia (p = 0,000) en comparación con los que siguieron el protocolo de vigilancia regular. Conclusión: Los programas educativos basados en el empoderamiento parecen contribuir a mejorar la autoeficacia percibida, por lo que pueden asumirse como estrategias para aplicar a los pacientes con diabetes mellitus tipo 2.