GRANT and AWARDS SERIES (The Michael J. Fox Foundation):Nicoletta Plotegher & Elisa Greggio

Pubblicato il: 23.11.2021 15:30

Using Nanobodies and Peptide Mimetics to Stabilize and Activate Mutant GBA

Defects in the stability and activity of the lysosomal enzyme GBA1 are associated to Parkinson’s disease etiology, in both sporadic and genetic forms of the disease. In a previous MJFF-funded project, we generated a special type of antibodies, called nanobodies, against GBA1 and studied their ability to stabilize and activate the enzyme in vitro. The goal of this new project is to understand the mode of action of GBA1 nanobodies by determining the 3D structure of the complex GBA1-nanobody and investigate their impact on GBA1 function in cellular and neuronal models for Parkinson’s disease. We expect to be able to improve GBA1 activity via nanobodies and exploit them as a novel valuable therapeutic approach.

PI's Biography

PI: Nicoletta Plotegher was originally trained as physicist and then decided to move toward biosciences for her PhD and postdocs. Her research is focused on the study of the molecular mechanisms of neuronal function and dysfunction. She is passionate about fancy microscopes and likes playing with numbers. Besides science, she loves reading books, cooking and travelling. 

Co-PI: Elisa Greggio is a molecular physiologist who trained at the NIH (USA) in the field of neuroscience and Parkinson’s disease during her postdoctoral studies. She has been studying for almost two decades the pathobiology of LRRK2, a kinase responsible for familial and sporadic forms of Parkinson’s disease and other immune-related disorders. In addition to science, she loves painting, yoga and adventure travelling. 

www.michaeljfox.org/grant/using-nanobodies-and-peptide-mimetics-stabilize-and-activate-mutant-gba