RNA therapy development for inherited eye diseases

Applications are welcomed for self-funded students to pursue a PhD in RNA therapeutics. The PhD project will use short activating RNAs to specifically increase target gene expression in inherited or acquired eye disease. The work will build upon extensive local expertise combined with input from the world-leader company on short activating RNAs (saRNA).

In recent years our work has focused on an inherited eye disease called aniridia. For most patients with aniridia, they are born with one functional copy of the PAX6 gene and one non-functional copy. Using saRNAs, we have sought to target the functional copy and increase expression of the protein as a means to rescue the disease. Our work to date has established the feasibility of this approach working from cells in a dish through to animal models. The next stage of the project will be to test efficacy in treating a mouse model of the disease (in collaboration with colleagues at the University of Aberdeen).

In addition, we have identified a number of alternative targets where saRNA technology could be used to improve patient outcomes. Therefore, we would also/alternatively welcome a PhD student to work on new targets for therapy development. Alternatively or in addition, projects can be developed using our existing tools to investigate gene/protein function with specific discovery science projects centred around alternative splicing and circadian regulation available to be pursued. We anticipate the final PhD that your pursue will be a whatever bespoke combination of the translational and discovery science parts to best suit your interest and career plans.

Ideally the successful applicant will have a strong background in genetics and practical experience in core cell and molecular techniques such as cell or tissue culture, RNA isolation and RT-qPCR. Note that these lab skills are not an absolute requirement, our research team are very happy to train candidates in everything they require. The work will primarily be performed in the state of the art labs at the University of Liverpool but will likely also spend time at the University of Aberdeen and/or the headquarters of MiNA therapeutics in London, or collaborators at the University of Ulster and at Wills Eye Hospital, Philidelphia.

Applications should be made to the project supervisors in the first instance via CV and cover letter. We will then discuss project options and what would be best suited to you. Note that only when a candidate has been selected will a formal online application be required.