It has been an exciting few months for research. In January, we reported the fantastic results of the gene therapy clinical trial currently underway in the University of Oxford for choroideremia, a rare retinal degeneration predominantly affecting men. Patients with a mixture of early and advanced stage choroideremia were treated with a viral vector coding for the CHM gene. Those with early stage disease didn’t experience any detrimental decrease in vision, but most encouragingly, two individuals with advanced stage choroideremia had substantial gains in their visual acuity. One man remarked that he could see the numbers on his mobile phone for the first time in five years.
Trials like this put our Target 3000 project in context. Target 3000 aims to identify the causative gene for inherited retinal disease in every affected individual in Ireland, enabling the first step to determining eligibility for cutting-edge clinical trials. Almost 300 individuals have been recruited so far, with genetic sequencing and correlating eye examinations on-going at the Royal Victoria Eye and Ear and Mater hospitals. This
accelerated recruitment has been achieved by our recent investment in hiring two clinical research fellows in the respective hospitals, and our continued support of the next-generation genetic sequencing research in conjunction with the Health Research Board. We are committed to our goal of recruiting many more people to Target 3000 in the coming months by working with our scientists, doctors and members across the country.
For more information about how you can get involved, please contact me on 01 6789 004 or firstname.lastname@example.org.
Dr Maria Meehan, Research Manager