2019 BIST Ignite Project: NANO-GBA

Assessing how glucocerebrosidase (GBA) defects alter receptor membrane nanoarchitecture to design improved nanomedicines

The NANO-GBA project received a 2019 BIST Ignite Grant at the annual BIST Ignite Awards Ceremony held in March 2020.

Gabby Silberman (BIST Director) presents the 2019 BIST Ignite Grant to NANO-GBA

Project overview

Alterations in the Glucocerebrosidase enzyme (GBA), which is essential for the metabolism of fats, are directly linked to rare diseases such as Gaucher’s disease, and to neurodegenerative disorders such as Parkinson’s. These alterations affect the cell membranes and the transport of natural substances or medicines from the blood to the brain. Silvia Muro, ICREA Professor at  IBEC and expert in drug delivery, and María García-Parajo, ICREA Professor at ICFO and specialist in nanophotonics, have teamed up for the first time to investigate the workings of these biochemical and biophysical alterations in the cell membrane, the mobility and nanoclustering of receptors on the cell, and the transport of substances across the blood-brain barrier. The ultimate goal is to better understand the progression of diseases like Gaucher’s or Parkinson’s, in order to design new strategies for diagnosis and treatment.

Multidisciplinarity within NANO-GBA

This multidisciplinary project encompasses molecular pathology, cellular biology, biotechnology, biochemistry, advanced optics, nanotechnology, and drug delivery. Muro (Targeted Therapeutics and Nanodevices-IBEC) is a biologist with expertise in drug delivery. She studies how cells transport cargoes to precise body destinations to design NCs and deliver therapeutics to specific disease-sites. She works with other lipidoses and BBB transport, not GBA-nanomedicines for Gaucher’s and Parkinson’s treatment or advanced nanophotonics. García-Parajo (Single Molecule Biophotonics-ICFO) is a physicist with expertise in nanophotonics and biophysics, who studies biological processes in living cells with single-molecule and nanoscale spatiotemporal resolution. She works on distribution of lipid domains/nanoclustering of immune-related adhesion molecules, not enzyme-caused lipidoses, BBB transcytosis, or targeted-NC design. Uniting their efforts for the first time has the potential for significant impact in this multidisciplinary research project.