Research Fellow in Therapy Development for Norrie Disease

at  University College London

ABOUT US

Our mission is to maximise and advocate for the holistic health of all children, young people and the adults they will become, through world-class research, education and public engagement. The UCL GOS ICH, together with its clinical partner Great Ormond Street Hospital for Children, forms the largest concentration of children’s health research outside North America. The 2024-29 GOS ICH strategy focuses on its five scientific programmes. GOS ICH’s activities include active engagement with children and families, to ensure that our work is relevant and appropriate to their needs. GOS ICH generates the funding for our research by setting out our proposals in high quality applications to public, charitable and industrial funding bodies and disseminates the results of our research by publication in the medical and scientific literature, to clinicians, policy makers and the wider public. The Institute offers world-class education and training across a wide range of teaching and life learning programmes which address the needs of students and professional groups who are interested in and undertaking work relevant to child health. GOS ICH holds an Athena SWAN Charter Gold Award.

We are seeking to appoint a highly motivated researcher with experience of AAV gene therapy to work in research on gene therapy to prevent hearing loss in patients with Norrie disease. Norrie disease is a rare genetic condition that primarily affects boys who are born blind and from about 12 years of age develop progressively worsening hearing loss. The post holder will evaluate NDP gene augmentation via a viral construct, and test its effectiveness in mouse models of Norrie disease. Candidates will investigate whether the gene therapy prevents death of the sensory hair cells in the cochlea of the inner ear and hearing loss, and restores retinal vascular barrier function. They will test whether restoring the missing NDP-induced intracellular signaling in endothelial cells prevents hearing loss. These studies will help design NDP viral constructs for clinical Norrie gene therapy. The post would suit a postdoctoral researcher with a strong background and practical experience in AAV therapies and interest in degenerative disease and therapeutic development. This post is supported by funding from Great Ormond Street Hospital Children’s Charity (GOSHCC), the salary offered is £42,099 - £45,521 per annum and is available from 1st December 2024 for 28 months in the first instance, with possibility for extension subject to funding availability.