Creating a MPSIII zebrafish model and brain specific in vivo lysosome reporter for drug screening and in vivo imaging of disease pathology

Project: Research

Project Details

Project Description

We propose to use our expertise in the zebrafish disease modelling, stem cells and tissue repair to create models to study MPSIII. The glycosaminoglycan metabolism pathway including the lysosomal enzymes that are causing MPSIII variants are fully conserved in zebrafish.
Effective start/end date1/02/1831/01/19