Funding outcome

Batten disease is a rare and devastating neurodegenerative disease that destroys neurons in the brain and causes death in children and infants. Currently, there is no cure or treatment to stop this disease. In Batten disease, damaged lipids and proteins accumulate in an organelle called a lysosome. Lysosomes are important for breaking down damaged and worn-out components of the cell. However, lysosomes stop functioning when they accumulate damaged lipids and proteins. Neurons in the brain are particularly vulnerable to lysosomal dysfunction and the accumulation of damaged molecules. We previously discovered two different strategies to promote the release of these damaged molecules from neurons. We can either promote the ability of neurons to empty the inner contents of the lysosome outside the cell, or treat neurons with a protein that pulls damaged molecules from neurons, preventing these molecules from getting to the lysosome in the first place. Both of these treatments protect neurons from death in models of adult neurodegenerative disease. This project will explore whether these strategies can similarly protect neurons in Batten disease. Ultimately, we hope to gain a better understanding of what contributes to neurodegeneration in Batten disease and to reveal new therapeutic strategies to treat children with this terrible disease.