Drug screening using Homer1 patient’s iPSCs-derived neurons for clinical trials of precision medicine

The Stem Cell Laboratory in the Department of Neuroscience is investigating new treatments for a class of patients that do not respond to mainstream anti-seizure medications (ASMs). Our laboratory has created patient-derived neurons for the purpose of creating an in vitro disease model to test the efficacy of these potentially life-changing novel ASMs. In this Honours project we will utilise the patient-derived iPSC-derived neuronal cultures with functional multi-electrode array (MEA) analysis to screen an FDA-approved drug library to identify drugs that can be readily repurposed into “n of 1” clinical trials of patients with Homer1 mutations. The project has the potential to identify effective “personalised” medicine treatment for patients with brain diseases due to Homer1 mutations. It also has the potential to provide new insights of the role of mechanisms related to the Homer family in neurobiological normal function and disease that may open up new treatment options for other related brain diseases. This project will require the techniques of stem cell-based in vitro cell culture, computer-aided drug designing, immunohistochemistry and gene expression analysis using real time PCR.