Objective: G-Can, the GBA1 Canada Initiative, is an open-science platform which aims to advance GBA1 research and develop treatments for GBA1 mutation-related Parkinson’s disease (PD). Leveraging open science and extensive partnerships, it also aims to establish trial-ready populations in preparation for future clinical trials.

Background: Mutations in the GBA1 gene, critical for lipid metabolism, are associated with Parkinson’s disease (PD), found in 5% – 20% of PD cases in different populations. GBA1 related research is a rapidly growing field in neurodegenerative disease studies. Current PD treatments focus on symptoms management rather than on the underlying causes, emphasizing a pressing need for research on GBA1 to develop targeted therapies.

Method: Currently, our research focuses on development and use of models (human cell lines, organoids and animals), data (clinical, imaging, genetic, transcriptomic) and research tools (assays, antibodies). These have been generated, all with a specific focus on GBA1. To further our research objectives, this platform will support other specific GBA1 themed research projects, including basic science, translational, and clinical research, through open requests for application.

Results: A total of 24 cell and animal models of GBA1 and its modifiers (Table 1) have been generated and are now available through C-BIG, an open science data infrastructure- https://cbigr-open.loris.ca/ipsc_catalogue/. A zebrafish GBA1 model has been generated using CRISPR to introduce the analogous mutation encoding the GBA1 [W378G] variant (Figure 1). We performed structural analysis on all single nucleotide variants in the GBA1-PD browser (https://pdgenetics.shinyapps.io/gba1browser/) using AlphaMissense (AM) pathogenicity scores (Figure 2). Lastly, antibodies were characterized (Figure 3), in vitro and in situ assays for measuring GCase activity have been developed and tested (Figure 4).

Conclusion: The models, tools and assays developed are accessible to the larger research community to support GBA1 research through C-BIG (https://cbigr-open.loris.ca/). By fostering data sharing and collaboration, G-Can strengthens research efforts and increases the potential for discovering innovative and effective treatments for GBA1-related diseases.

Table 1

Figure 1

Figure 2

Figure 3

Figure 4

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MDS Abstracts - https://www.mdsabstracts.org/abstract/g-can-the-gba1-canada-initiative/