Research Assistant

About the role A Research Assistant position is available in the laboratory of Professor Julien Baruteau at the Great Ormond Street Institute of Child Health. The successful candidate will join a GOSH Charity-funded project developing mRNA therapy for neuronopathic Gaucher disease, a severe and currently incurable form of Gaucher disease characterised by progressive neurological involvement. The project aims to develop a therapeutic mRNA approach capable of restoring glucocerebrosidase (GCase) activity in the brain and ultimately providing a new treatment strategy for patients with neuronopathic disease. The project is already progressing well, with optimisation of the therapeutic mRNA construct underway and promising initial in vitro results. Over the course of the 12–15-month appointment, the post-holder will play a key role in advancing the programme towards in vivo proof-of-concept. Their responsibilities will include: • Completing the in vitro optimisation and characterisation of the therapeutic mRNA construct. • Investigating mRNA delivery to the mouse brain and evaluating expression and biodistribution. • Maintaining and managing a mouse model of neuronopathic Gaucher disease. • Performing in vivo studies to assess the therapeutic efficacy of the optimised mRNA construct. • Contributing to experimental design, data analysis, interpretation and dissemination of findings. The post-holder will benefit from close scientific interaction with Professor Ahad Rahim at the UCL School of Pharmacy and Professor Stephen Hart at UCL Great Ormond Street Institute of Child Health, providing an exciting multidisciplinary environment spanning RNA therapeutics, gene therapy, neuroscience and rare disease research. About you We are seeking a highly motivated scientist who is enthusiastic about developing innovative therapies for children affected by rare neurological diseases. Candidates should have recently obtained, or be about to obtain, an MSc in Neuroscience, Molecular Biology, Biomedical Sciences or a closely related discipline. Experience with molecular biology techniques and rodent models is expected, together with strong skills in scientific communication, literature review and scientific writing. Previous hands-on experience in mRNA research and technology is essential. This is an excellent opportunity to join a dynamic translational research programme and contribute to the development of a potentially transformative therapeutic approach for neuronopathic Gaucher disease. Post is available for 12 -14 Months. Customer advert reference: B02-11319