Cameron McKnight

Childhood mitochondrial disorders Professor David Thorburn
PhD student at the University of Melbourne
Recipient of scholarship
I am working on running a pre-clinical drug trial of potential therapeutic interventions for childhood mitochondrial disorders. I am using cardiomyocytes and neurons generated from gene knockout and corrected pluripotent stem cell lines I have generated using CRISPR-Cas9 to study the tissue specific effects of treatments. I am working with both hESC and iPSC lines in this project and using a range of techniques to look at cellular phenotypes, including calcium imaging and MEAs.

My future research directions include: generation and functional study of induced pluripotent stem cells from mitochondrial disease patients; further investigation of tissue specific phenotypes associated with mitochondrial disease gene knockouts via differentiation to more clinically relevant cell types; and screening of potential drug treatments.