Purpose of the STSM: The main purpose of this STSM was to learn all the steps of the CRISPR-Cas9 technique applied to the generation of a knock-out zebrafish model, as well as an introduction to various phenotyping approaches. Within the scope of Maria Francisca Coutinho’s ongoing project “Neurological disease modelling for Sanfilippo: a key step towards understanding and treating a rare genetic disorder” (EXPL/BTM-SAL/0659/2021), which aims to create knock-out zebrafish models for Sanfilippo to be used in the research for new RNA-based therapies, it was extremely important for the applicant to learn and perform this technology and then bring the knowledge and transfer it to other colleagues from Sandra Alves’ lab on Lysosomal Storage Disorders (Research and Development Unit, Human Genetics Department from the National Institute of Health Dr. Ricardo Jorge, in Portugal).

Furthermore, an additional objective was to take advantage of the STSM period not only to learn the whole method but also to start the generation and characterization of a zebrafish model for Sanfilippo disease.

Grantee name: Luciana Maria Vaz Moreira

STSM start and end date: 02/10/2022 to 27/10/2022

Title: CRISPR-Cas9 in zebrafish for generation of a knock-out model for MPS III (Sanfilippo Syndrome)
Host lab: Prof. Stephen Wilson, University College London (UCL) Department of Cell and Developmental Biology (London, GB)