Amaral, OlgaDuarte, A.J.2017-02-212017-02-212016-07Olga Amaral; Foreseen accomplishments through precision cellular and molecular biology, 14th international onference on rare diseases "rare diseases: our task", July 2016, Bialobrzegi-Polóniahttp://hdl.handle.net/10400.18/4342Lecture by Olga Amaral - invitation by the organizing committee.LSDs therapies generate large expenses, having a major effect on health systems and family resources. It is, therefore inevitable to attempt to develop effective and economically viable models and therapeutic approaches. Through the development of specific cellular models, we hope to contribute to the increase of choices in terms of therapeutic interventions. Generation and application of iPS cells for the establishment of cellular models for lysosomal diseases has been applied to a few disorders. This approach can allow easier access to disease-specific cells, which will retain the original genotype. We expect that its application to LSDs will provide the means to study pathogenic mechanisms and therapeutic options.engDoenças GenéticasLysosomal DiseasesRare DiseasesMolecular TherapiesCell ModelsiPSCsconference object