Seeing Beyond Lecture Series October 2019: Ocular gene and call therapy: from bench to bedside and beyond
On Wednesday the 2nd of October Professor Keith Martin and Doctor Lauren Ayton presented at the Department’s Seeing Beyond Lecture Series. Thank you to all who attended (and rural practitioners who joined us online). We look forward to your attendance in 2020!
Professor Keith Martin
Gene therapy promises to revolutionise the treatment of inherited eye disease. To date, this treatment has only been applied to rare diseases caused by single gene defects. Glaucoma is the leading cause of irreversible blindness worldwide, but only a small minority of glaucoma cases have single gene defects. In this lecture, Professor Martin discussed these ideas as well as the different challenges that arise when treating more complex eye diseases. Professor Martin also gave an overview of the current ocular gene therapies, and a glimpse into their future possibilities.
Dr. Lauren Ayton
Ocular gene therapies are rapidly moving from preclinical experimentation to commercially-available clinical treatments. In late 2017, the first ever direct-to-patient gene therapy was approved for Leber Congenital Amaurosis, a form of retinitis pigmentosa. Other treatment modalities are close behind, including gene editing technologies such as CRISPR. Dr Ayton described a clinically-relevant overview of the technologies, and a discussed the ocular diseases which may be amenable to treatment. In addition, she detailed the importance of optimal patient selection and sensitive outcome assessments in gene therapy clinical trials.