Leonard Petrucelli, Ph.D.

Our major research goals are to identify the processes that cause neurodegeneration in diseases characterized by abnormal protein aggregation and to develop therapeutic strategies for their treatment. Such diseases include Alzheimer's disease, frontotemporal lobar degeneration, amyotrophic lateral sclerosis and Parkinson's disease.

In order to more clearly understand the function of proteins implicated in disease progression, as well as the mechanisms responsible for causing neuronal death, we have developed cell and animal models that recapitulate key features of these diseases. These models are instrumental in gaining insight regarding the neurotoxic processes that initiate the abnormal aggregation of certain proteins, such as tau and TDP-43, as well as the harmful consequences resulting from the formation of proteinaceous aggregates. Concomitantly, we are committed to developing and discovering new therapeutics to target and treat these devastating conditions.

Therefore, we routinely employ in vitro, in vivo and molecular approaches to identify, improve and examine small-molecule and biotherapeutic drug candidates. Moreover, we are developing and characterizing novel biomarker assays which, in addition to their diagnostic value, are expected to provide a reliable and sensitive means to test drug efficacy and, ultimately, help determine the best course of action for patients once treatments become available.

Overall, our studies are geared toward deciphering the mechanisms of neurodegeneration in proteinopathies and translating that knowledge into the development of therapeutics.