Incubator Grants

Could Stem Cell-Derived Tissue Cultures Replace Animal Models for Drug Screening in Primary Mitochondrial Disease?

Vijay Rajagopal

Researcher

Dr Vijay Rajagopal

Research Institution

University of Melbourne

Project Name

Could Stem Cell-Derived Tissue Cultures Replace Animal Models for Drug Screening in Primary Mitochondrial Disease?

Project Details

Mitochondrial disease patients present a wide range of symptoms and across different organ systems that make it difficult to diagnose and treat the disease.

Currently, each patient with mitochondrial disease requires a tailored treatment approach. Treatment advice has largely been based on anecdotal evidence and a limited number of studies.

This project aims to verify whether stem cell-derived cardiomyocytes (cardiac muscle cells) are adequate model systems to mimic the function of adult ventricular myocytes. The analysis will enable researchers to customise stem cell-derived organoids for drug screening studies for patients with mitochondrial diseases.
Dr Rajagopal and his team will create computational models of the mitochondrial function. Using those models, Dr Rajagopal will define various mechanisms and determine whether the observation in the cardiac organoid can be translated to the adult cardiac muscle cell phenotype.

This strategy could drastically reduce the time from research to bedside by removing the need for animal models and accounting for the patient-specific disease condition during drug testing.