MSc in Biological Engineering, Instituto Superior Técnico, University of Lisbon, Portugal, 2021
My project focus on using hESCs with targeted genetic engineering mediated by TALENS, a system that allows targeted genetic manipulation of the genome. For that, a targeting strategy will be designed to introduce inducible forms of fluorescently labelled dead-Cas9 (dCas9) fused to transcriptional activators (e.g. VPR) or repressors (e.g. KRAB) domains, into a defined locus in the human genome. This strategy will be used to engineer two well characterized hESCs, H1 (male) and H9 (female) as cellular tools for disease modelling that can be tested by designing guide RNAs to target several genes, for either repression or activation, like MECP2 and UBE3A, which mutations are associated with Rett and Angelman neurodevelopmental Syndromes, respectively.