Scientific Strategy
Steadfast in our mission to foster and accelerate innovative treatments and a cure for PolG disorders, The PolG Foundation aims to be a conduit for scientific collaboration by connecting academic research, drug discovery and clinical development together.
To achieve our PolG research goals, we work in several different ways:
We support PolG academic researchers around the world by funding a diversity of preclinical, translational and clinical initiatives.
PolG iPSC Cells and Mouse Model available. See our PolG Toolbox items and contact us for further details.
We have funded the development of two PolG Transgenic Mice. Characterisation is underway.
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
