Project Code
CMD27Br Saleem
Project Type
Wet lab
Research Theme
Cardiometabolic Disease
Project Summary Download
Summary
Cystinosis is a rare condition caused by mutations in the CTNS gene. This leads to cystine build-up in cells which damages many organs, especially the kidneys. The current treatment, cysteamine, slows the disease but many patients still develop serious complications. This project aims to develop a new, one-time gene therapy that provides a lasting treatment for cystinosis, by integrating a healthy copy of the CTNS gene into cells. We will test whether this reduces the harmful cystine build-up. If successful, this work could lead to a single treatment that prevents serious multi-organ complications and improves quality of life in cystinosis.
Can the project be completed part time?
Yes
Lead Supervisor
Professor Moin Saleem
Lead Supervisor Email
University Affiliation
University of Bristol
