Physiopathology studies and therapeutical approaches in animal and cellular models of neurometabolic diseases

 The research group (www.cbm.uam.es/lab220 ) works generating and characterizing animal and cellular (iPSC) models relevant for specific diseases, to be used as research tools to understand the molecular and physiopathological mechanisms responsible for disease,  to analyse potential biomarkers for prognosis and follow-up and to identify new therapeutical targets. The ultimate aim is to develop personalized RNA based therapies (antisense oligonucleotides) as well as pharmacological therapies with antioxidant compounds and mitochondrial activators, performing preclinical studies in the specific disease models. The student will work on one of the following projects: differentiation of iPSC derived from patients' fibroblasts into relevant cell lineages (astrocytes, neurons, cardiomyocytes...) or into brain organoids; use of gene editing techniques to  generate cellular models with specific splicing mutations, to identify and test candidate therapeutical antisense oligonucleotides; characterization of new mosue models of neurometabolic disease at the molecular and biochemical level. The project will cover different aspects within these topics using several techniques  such as bioinformatics analysis, cell culture, confocal microscopy, Seahorse analysis, transfections, RT-PCR, Western blot analysis,CRISPR/Cas, base editing, prime editing, mouse neurological phenotyping…

We are looking for motivated students who would like to pursue a scientific career doing a PhD after the Master.