Centre for Rare Diseases & Gene Therapy
Guiding therapies for rare diseases.
About the Centre
At the Centre for Rare Diseases & Gene Therapy, we investigate the fundamental mechanisms of gene regulation to drive new treatments for rare inherited metabolic and blood disorders, cancer and chronic inflammatory disease.
Our team is developing next‑generation adeno‑associated virus (AAV) gene therapies engineered for safer, more efficient delivery at lower doses. By reducing dose‑related toxicities and immune‑driven side effects, we aim to deliver more effective, targeted therapies that genuinely improve outcomes for people living with rare diseases.
Laboratories
Bailey
Gene regulation is frequently dysregulated in cancer. During cancer initiation, transcription factors can exhibit somatic mutations including point mutations in regulatory domains or undergo copy number variations due to genetic deletion.
Student opportunities
Targeting Neuroinflammation with Novel Epigenetic Drugs
Projects: Honours, Masters & PhD
Project details
Primary Supervisor: Dr Mehdi Sharifi Tabar
Supervisory Team: Dr Chuck Bailey
Centre: Rare Diseases & Gene Therapy
Central nervous system-related autoimmune diseases, such as Aicardi-Goutières syndrome, and systemic conditions like Sjögren’s syndrome and systemic lupus erythematosus, are driven by the persistent overactivation of interferon-stimulated genes (ISGs). This persistent ISG activity causes inflammation and can lead to brain tissue damage. Currently, there are no effective treatments, highlighting an urgent need for innovative therapies. Our recent research has identified TRIP (Transcriptional Regulator of the Interferon Pathway) as a key epigenetic regulator of neuroinflammatory ISG expression, making it a highly promising therapeutic target for these diseases.
In this project, using a multidisciplinary approach, combining molecular, cellular, biochemical, and genetic experiments, we aim to examine two new immunoregulatory drugs that selectively target TRIP mRNA and protein to suppress neuroinflammatory ISGs.
* For intellectual property reasons, we have named the gene TRIP
Get in touch
To get in touch and for all general enquiries relating to our work, please contact Dr Charles Bailey.