Cambridge Institute for Medical Research
Lay Overview of the project:
Multiple Sulfatase Deficiency (MSD) is currently an untreatable disease. From studies performed on cells and on samples from patients, we now know some of the processes inside cells that cause or influence the disease. While there is a lot to be gained from studying simple cell culture systems, this does not tell us about how the disease affects different tissues (e.g. nerves compared to muscles) and whether defects in one tissue can have consequences for other parts of the body. For this reason, we need to develop good animal models that share the same features of the disease as we see in patients. We are generating a zebrafish model for MSD that will be used for such purpose. Zebrafish are small tropical fish that are popular as a model for studying the causes of different diseases and for screening compounds to find new drugs to treat these diseases. They are vertebrates and so have very similar tissues and organs as man. Their whole genome has been sequenced and so we know the similarity between the genes and proteins in zebrafish and man. For more information on zebrafish, click here(https://irp.nih.gov/blog/post/2016/08/why-use-zebrafish-to-study-human-diseases). We will study MSD in our zebrafish model and perform a screen to identify compounds which improved the disease in the zebrafish and so therefore may be suitable to treat the disease in man.