Andrew Nguyen

Our lab focuses on progranulin – a protein linked to frontotemporal dementia (FTD) – and how its deficiency contributes to neurodegenerative and metabolic diseases. We are currently investigating progranulin's structure and function using a variety of molecular and cellular approaches. We are also testing strategies (including antisense oligonucleotides) for increasing progranulin levels as potential therapies for progranulin-deficient FTD. For this work, we are focusing on the progranulin R493X mutation, which is the most common progranulin mutation found in individuals with FTD, using a knock-in mouse model as well as patient-derived fibroblasts.