Aimee Kao, MD, PhD
University of California San Francisco
TDP-43 mutations and lysosomal proteolysis
The Kao Lab is interested in the basic mechanisms driving development of neurodegenerative diseases like amyotrophic lateral sclerosis (ALS) and Alzheimer’s Disease (AD). Our work has led us to focus specifically on the lysosome, a part of the cell responsible for degrading and clearing away neurodegenerative disease proteins such as TDP-43 and tau. Here, we propose to investigate if tuning lysosomal activity up or down affects clearance of TDP-43 in neurons and can be considered as a therapeutic avenue in ALS.