Maria Francisca Coutinho is an Auxiliary Researcher at the National Institute of Health Dr. Ricardo Jorge in Portugal and member of the FCT Research Unit, Center for the Study of Animal Science (CECA/ICETA, University of Porto) that now integrates the new associate laboratory AL4animals. Ever since she finished her degree in Biology, in 2007, her scientific activity has been focused in the field of Health Sciences, particularly in the Human Genetics area. She has been working in Lysosomal Storage Disorders (LSDs), one of the major groups of genetic disorders, affecting both children and adults. Over these years she has actively contributed to the development of new methods for diagnosis and characterization of LSD patients and to the study of the pathophysiological mechanisms of these diseases. More recently, though, she has also been involved on a number of proof-of-principle studies to design RNA-based therapies aimed at correcting or ameliorating the cellular phenotype of a few LSD. While exploratory, these studies have highlighted the two major obstacles that may hinder the clinical translation of this sort of approaches: the need for adequate delivery vectors and suitable disease models. Currently, she is addressing this second concern, by running a project that relies on development of in vitro and in vivo disease models for MPSIII, a severely debilitating neurodegenerative LSD of infantile onset. Briefly, two different workflows are ongoing. First, we are establishing an innovative approach to generate patient-derived MPSIII neuronal cell lines through a non-invasive approach using dental pulp stem cells (DPSC). For the in vivo model, we are generating zebrafish (Danio rerio) models using the CRISPR/Cas9 system to generate stable mutant lines for each MPSIII-associated gene. Ultimately, those models will constitute a great platform to uncover novel disease-related pathways and yet unknown drug targets for this challenging group of disorders. Finally, in an era where personalized medicine and mutation-specific therapeutic approaches are gaining momentum, they will also constitute optimal platforms for in vitro drug testing.