MDS 2021 | Precision medicine in GBA-associated PD: targeting the underlying molecular pathways

Precision medicine approaches in glucocerebrosidase (GBA)-associated Parkinson’s disease (PD) will enhance treatment specificity by targeting the specific GBA variant and associated molecular pathways. Enza Maria Valente, MD, PhD, IRCCS Mondino Foundation & University of Pavia, Pavia, Italy, discusses some approaches under investigation, such as increasing the levels of the GBA enzyme in the presence of truncation mutations which would abolish the production of functional GBA protein. In other cases, despite the production of protein, the mutated form may have reduced activity or be unable to move to the lysosome where it acts. In this case, Prof. Valente explains that chaperone proteins may be used to enhance transportation. This interview took place during the 2021 International Congress of Parkinson’s Disease and Movement Disorders.