Dopamine in Lesch-Nyhan Disease
Awardee: Jasper Visser
Institution: Radboud University Medical Center, Nijmegen, The Netherlands
Grant Amount: $85,779.00
Funding Period: February 1, 2023 - January 31, 2024
Summary:
In Lesch-Nyhan disease (LND), it is unknown why the loss of a very generic protein that is present in all cells of the body causes such a specific brain dysfunction with abnormal movements and self-injury. It is thought that brain cells that use the neurotransmitter dopamine to pass on signals to other brain cells are particularly affected in LND, but it is not known whether these are the only brain cells that are affected. This project investigates in a mouse model for LND whether repairing these dopamine neurons alone would be sufficient to prevent or treat the brain abnormalities in LND. If that is the case, they could serve as the primary target for future therapies.