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

Project: Research

Project Details

Project Description

The Homer family of proteins are believed to be involved in glutamatergic excitatory neuronal signalling, but their mechanisms of neurophysiological action, and role in the pathophysiology of brain diseases such as autism, intellectual disability, epilepsy and autoimmune encephalitis, are currently poorly understood. Recently, a patient with this phenotype has been identified with a mutation in Homer1, and neuronal cultures have been generated from their blood derived induced pluripotent stem cells (iPSC). This proposal aims to utilize these 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.
StatusActive
Effective start/end date17/04/2330/09/24

Funding

  • Homer Hack Ltd: A$40,000.00

Keywords

  • Precision Medicine
  • In Vitro Drug Screening
  • Stem Cell-based disease model
  • Neurodegeneration