Gene Therapy Fact or False Dawn

Prof Lyndon da Cruz

Meeting:  2017 RANZCO


Date:      -

Session Title: Plenary Session

Session Time:      -

Speakers and Topics:
Gene therapy entered the clinical realm in the early 90?s following the molecular biology revolution. The ability to harness and manipulate virus vectors and to use other mechanisms to introduce genes into cells in vivo promised a new generation of treatments for inherited monogenic disease as well as disease processes such as in?ammation and neovascularization. Almost 25 years later there is not a single gene therapy treatment widely available for ocular use and it certainly has not revolutionised the treatment of any group of diseases or processes. Was it all hype or where the expectation too high for something that has in an appropriate time frame brought a powerful new treatment paradigm to the clinic. The answer is somewhere in between and the arrival now of many trials both in inherited and non-genetic ocular diseases may herald the long-awaited arrival of this ?new treatment?.