Piera Pasinelli, PhD
Frances & Joseph Weinberg Professor of Neuroscience, Department of Neuroscience
Director, Jefferson Weinberg ALS Center
Vickie & Jack Farber Institute for Neuroscience
RESEARCH & CLINICAL INTERESTS
Research in Dr. Pasinelli’s laboratory in the Weinberg Unit for ALS Research focuses on the study of the cellular and molecular events that lead to motor neuron death in Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig’s disease.
ALS is a typical neurodegenerative disease caused by degeneration and death of motor neurons in the spinal cord, brain stem and motor cortex. This leads to muscular atrophy. Death occurs 3 to 5 years from onset. Currently there is no cure for ALS.
The work done in the laboratory focuses on the molecular mechanisms of motor neuron degeneration caused by two ALS-associated proteins. Disease causative mutations in these proteins cause them to undergo conformational modifications and mislocalize within the cells. The goal is to understand the correlation between toxicity and mislocalization and to identify the molecular targets of these mislocalized proteins once they reach the cellular compartment were they do not normally reside. The ultimate goal is to identify potential therapeutic targets. To this end, work in the laboratory develops in two components: (1) Basic research and (2) Translational research.