Funding outcome

Batten disease is a devastating and fatal neurodegenerative disease that affects children. It involves the accumulation of damaged lipids within an organelle called a lysosome. Lysosomes are needed to break down worn-out components of cells. However, when they accumulate damaged lipids, they stop working properly. Neurons in the brain are particularly vulnerable to the accumulation of damaged lipids and defective lysosomes. We previously discovered multiple strategies to promote neurons to release damaged lipids. This improved neuron health in models of adult neurodegenerative disease. In this project, we will explore whether these strategies can be used to protect Batten disease neurons. Specifically, we will test if we can reduce the accumulation of damaged lipids, and prevent neuron death. Ultimately, we hope to gain a better understanding of what contributes to the degeneration of Batten disease neurons and to reveal new therapeutic strategies to treat children with this terrible disease.