New chemical approaches to improve A-to-I RNA editing with the aim of restoring sight loss associated with oculocutaneous albinism (OCA)

In this project, we will evaluate whether chemically modified nucleic acids can be used to restore melanin production in OCA, potentially reversing sight loss associated with the disease. Unlike conventional drugs which act on proteins, nucleic acid therapeutics target the genetic blueprint of a disease and are emerging as a powerful new class of precision medicines. We will use organic chemistry to design and synthesise new chemical modifications and evaluate whether these enhance the activity of nucleic acid therapeutics. We will target a common pathogenic variant which the Self group have identified as a significant contributor to OCA. Whilst this project focuses on developing nucleic acid therapeutics for OCA, the chemical modifications generated are likely to be applicable to many other diseases, including rare and those currently considered 'undruggable'.

This is an interdisciplinary project, and you will receive training in a range of organic and chemical biology techniques during the studentship. These will include synthetic organic chemistry, solid-phase oligonucleotide chemistry, biophysical and biochemical techniques, and biological assays in cell culture.