Duarte, Ana JoanaBragança, JoséAmaral, Olga2018-02-022018-02-022017-05http://hdl.handle.net/10400.18/4927Work carried out at Centro de Saúde Pública Dr. Gonçalves Ferreira (Porto) and Universidade do Algarve- Faro (supervision of Prof. Bragança).The lack of good disease models limits the understanding of the human pathophysiologic mechanisms and hinders investigation research and development of new therapies. In 2006, Yamanaka’s group expressed four transcription factors (Oct4, Sox2, Klf4, and c-Myc) producing induced pluripotent stem cells (iPSCs), allowing the development of new strategies for pathogenesis modeling and drug testing. iPSCs generated from somatic cells from patients are a desirable source for patient-specific studies since they maintain the patient’s genetic background. In this work, we ultimately aim to develop induced pluripotent stem cells (iPSCs) from Lysosomal storage disorders (LSDs) patient’s fibroblasts and normal controls to produce disease models. Thus, using iPSCs methods to generate the cell-targets to reproduce the disease may create an ideal model for studying pathogenic mechanisms. The initial biological material being used consists of commercially obtained human control dermal fibroblasts. This type of material guarantees better consistency in technical conditions. We are testing two different non-integrative polycistronic plasmid vectors in order to achieve forced expression of the Yamanaka’s transcription factors. For this achievement, transformation conditions with different vehicles of delivery were tested: different transfection reagents, concentration ratios, and timings were compared. Since we are beginning this work from zero, only a few very preliminary results were obtained and will be presented.engStem CellsiPSCGenética HumanaDoenças Genéticasconference object