Principle Investigator: Dr John Counsell, Associate Professor, University College London
Researchers at UCL are working on two promising gene therapy treatments for MSUD. The first method uses an optimised gene delivery technology to treat the liver, with the aim of permanently restoring functional metabolism in liver cells for the rest of the patient’s life. The second approach focuses on gene delivery to the brain, to investigate whether restoration of metabolism throughout the brain can alleviate neurological symptoms of MSUD better than treating the liver alone.