During Michaelmas Term, OxTalks will be moving to a new platform (full details are available on the Staff Gateway).
For now, continue using the current page and event submission process (freeze period dates to be advised).
If you have any questions, please contact halo@digital.ox.ac.uk
Based on the most recent estimates, there are over 10,000 known human diseases, and more are being discovered each day. A large fraction of these are rare monogenic (Mendelian) disorders. Gene-targeted therapies, such as gene therapy, gene editing, and oligonucleotides are therapeutic platforms that are broadly applicable to a large fraction of monogenic diseases. However, these technologies are currently developed as treatments for one disease at a time. This approach favors the most common rare diseases, and will leave many diseases and patients who could greatly benefit from gene targeted therapies behind. To address this disparity, a fundamentally different way of thinking about the problem is required. One potential solution, based on the science itself, is to develop gene-targeted therapies as therapeutic platforms to treat monogenic disease, rather than as treatments for one disease at a time. I will discuss the implications and challenges of this approach and provide examples of ongoing research in this area. See pave-gt.ncats.nih.gov ; fnih.org/our-programs/AMP/BGTC ; commonfund.nih.gov/editing .