To achieve our PolG research goals, we work in several different ways:
We have funded the development of two PolG Transgenic Mice. Characterisation is underway.
A Systematic Review of Animal and Cell Models of PolG-related Disease is awaiting publication.
We actively invite biotech and pharmaceutical companies to reach out and partner with us to develop PolG therapeutics.
Defined Areas of Research
Basic science: understanding mechanisms and modelling PolG disease
- Understanding the pathophysiology and how this can be manipulated for therapeutic intervention
- Generating models that mimic human disease. Special focus on mouse and stem cell models
- Using models to understand disease mechanisms
- Using models to test drugs/compounds
Getting ready for clinical trials
- Register/cohort studies
- Identification of genetic/environmental triggers
- Understanding the clinical diversity of this monogenic disease
Clinical trials including drug repurposing initiatives
- Pilot studies
- Full trials