The negative socioeconomic impact of blindness is amplified in patients with inherited retinal diseases in contrast to patients suffering from age-related visual impairments (such as macular degeneration) because of the early age at onset, visual challenges during educational years, and loss of visual function during prime working years. Retinal genetic disorders can now be targeted with genome editing techniques, but these need extensive optimisation and testing prior to clinical trial to ensure they are both safe and effective.
The Wellcome Discovery Award is designed to facilitate bold and creative research ideas and deliver significant shifts in understanding that could improve human life, health and wellbeing. The 4-year research programme funded by this £1.4M award will explore the basic mechanisms of gene editing in the retina using CRISPR based approaches, utilising novel transgene delivery systems previously developed in Professor MacLaren’s laboratory. These discovery-based scientific investigations will provide insight into the mechanisms of endogenous gene correction and the regulation of ectopic gene expression in photoreceptor cells, and may pave the way to improved therapeutic interventions for inherited retinal diseases.
Professor MacLaren said:
“I am delighted to have been successful with this award which will enable us to advance our promising CRISPR gene therapy programme into clinical trials.”