Applying specialized expertise and deep knowledge of lysosomal biology to treat neurodegenerative diseases.

A New Frontier in Neurodegeneration

Recent advances in our understanding of the biology underlying neurodegenerative disease highlight the import role lysosomes play across multiple disease states.

A Potential for Real Impact

Healthy lysosome function is critical for cellular recycling, homeostasis, and maintaining normal neuronal function. Across multiple nervous system cell types, lysosomal dysfunction can lead to substrate and protein accumulation, inflammation, and cellular damage that contributes to neurodegenerative disease.

Understanding the Underlying Biology

Lysosomes are intricate organelles in cells that play a central role in maintaining cellular health, especially in the cells of the central nervous system. Lysosomal dysfunction and the resulting disruption of protein homeostasis has been linked to the development and progression of a range of neurodegenerative diseases including Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis (ALS) and frontotemporal dementia.

Our team has discovered novel insights into the cellular processes regulating the delivery of proteins, such as progranulin, prosaposin and cathepsins, required to support proper lysosomal function.

A Promising Opportunity