https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/about-our-biomedical-research-centre/our-research-themes/accelerating-novel-therapies/
Accelerating novel therapies
Theme Leads: Professor Manju Kurian and Professor Darren Hargrave
Deputy Leads: Dr Karin Straathof and Dr Haiyan Zhou
Junior Faculty Representatives: Dr Wendy Haywood and Dr Karin Tuschl
Accelerating novel therapies develops treatments for rare diseases through early bench-to-bedside translation, innovative trial methodologies and biomarker development.
Working with over 180 different patient disease groups at GOSH, the theme brings together key skills in:
- Developing medicines that use RNA (ribo nucleic acid, a messenger molecule that carries instructions from DNA).
- Modelling diseases using human stems cells to 'grow' models of human disease in the brain, eye, skin, liver, kidneys and muscular system.
- CRISPR-based (gene-edited) screening platforms.
- Finding new targets for disease using proteomics (the study of someone's proteins) and genomics (the study of someone's genome or DNA).
Lead researchers in this Theme are running 85 phase one or two trials and 24 are in set-up.