- Country: Australia
- Official Title: Professor
- Department: Cell & Molecular Therapies
- Institute: Royal Prince Alfred Hospital
- E-Mail: jejrasko@gmail.com
Speech Title
Opportunities for Treating & Curing Disease in the Era of Gene Therapies
Professor Rasko’s talk examines the transformative potential of cutting-edge gene therapies, shedding light on the latest developments, breakthroughs, and future prospects. His presentation will offer valuable insights into the rapidly evolving landscape of cell and gene therapies and their promise in revolutionizing healthcare.
Gene modification can be achieved through two primary methods to be outlined: in vivo and ex vivo. In vivo gene therapy involves injecting genes directly into the body, potentially targeting tumours or other tissues. Ex vivo gene therapy involves removing a patient’s cells, modifying them outside the body, and then reintroducing them to the patient.
Gene therapy has made significant strides in treating haemophilia, thalassaemia and some other rare genetic diseases. In the case of haemophilia, by providing a one-time treatment that allows patients to produce necessary clotting factors themselves, gene therapy reduces reliance on ongoing treatments and avoids risks associated with blood product transfusions. Over the past 20 years, Professor Rasko has pioneered advancements in this field that have led to approved therapies for both haemophilia A and B in major markets.
The future of gene and cell therapy is promising. The ability to modify genetic material and reprogram cells offers the potential to address a wide range of diseases. While ethical, regulatory, and economic considerations are critical, the progress in this field suggests that gene and cell technologies could one day provide cures for many currently incurable conditions, offering hope for a brighter future in medicine.