£1 million award to push forward regenerative medicine research

Sterile GOSgene laboratory
Researchers at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health (ICH) are to lead a new collaboration exploring how stem cells can be engineered to evade immune responses. 

They will apply the latest genome editing technologies to modify ‘pluripotent stem cells’ – cells that can go on to become almost any cell in the body and could eventually be used to repair and regenerate diseased organs. There are around 6400 people waiting for organ transplants in the UK, and these new techniques may help some of them while they wait for suitable donors.

The project has been awarded a prestigious £1 million grant from the Medical Research Council (MRC) and is led by National Institute for Health Research (NIHR) GOSH Biomedical Research Centre investigators,  Waseem Qasim and Paolo De Coppi, both NIHR Professors at ICH and consultants at GOSH, as part of a collaborative network involving clinicians and scientists from regenerative medicine hubs across the UK.

The 3-year project aims to efficiently and safely test manufacture of 'universal cells' using cutting-edge CRISPR-Cas9 genome editing tools to make precise changes to the cells' DNA and disrupt ‘flags’ om the cell's surface called Human Leukocyte Antigens (HLAs). These flags are how the immune system usually distinguishes between ‘self’ and ‘non-self’ tissue and the project aims to deliver 'universal' cells that can evade immune rejection. State of the art sequencing and computer modelling will be used to assess the efficiency of the editing process and screen for unwanted effects. 

This new application of genome editing technology builds on strategies where immune cells are engineered to fight certain types of childhood cancer, and is part of wider initiatives to bring new types of treatments to NHS patients.