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Genes run just about everything that happens in our bodies, including how proteins behave in the brain. Too little or too much of a particular protein can cause some of the most devastating neurological diseases and disorders.

Dr. Brian Chen, a scientist from the Brain Repair and Integrative Neuroscience (BRaIN) Program at the (RI-MUHC), has been awarded the first Healthy Brains for Healthy Lives (HBHL) Neuro-Partnerships grant to develop a drug screening protocol designed to quickly identify promising treatments for patients with diseases caused by low gene expression.

The protocol will use a gene editing technique to insert a Protein Quantification Reporter (PQR) into a mutant gene to measure protein synthesis over time. Once the cells have been edited with the PQR, researchers will 鈥渨ash鈥� them with medical compounds, monitoring which ones boost protein synthesis.

Initially, Dr. Chen鈥檚 work will focus on three genes, including GBA1, which has been linked to Gaucher鈥檚 disease and Parkinson鈥檚 disease, but the drug-screening protocol could have broader applications.

鈥淭his approach will generate molecular treatments for any disease or disorder caused by insufficient gene expression,鈥� said Dr. Chen, 鈥渁nd open up a new avenue for individualized and rapid drug discovery for these patients.鈥�

Funds provided by HBHL and other partners will allow Dr. Chen鈥檚 lab to access libraries of FDA-approved compounds, and automate听high-throughput cell assays that will accelerate the pace of research.

鈥淚deally, we will find compounds that will allow us to skip directly to Phase 2 clinical trials,鈥� said Chen.

贬叠贬尝鈥檚 Neuro-Partnerships program co-funds projects with a high potential impact on biopharmaceutical research through existing programs offered by the Minist猫re de l'脡conomie et de l'Innovation (). MEI co-funding is available through Quantum Leap and SynergiQc programs, which includes funding from Pfizer Canada. The Brain Canada Foundation has also contributed to Dr. Chen鈥檚 research.

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