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Genes to cells,

& cells to patients

 

Our university research group, based at Great Ormond Street Institute of Child Health, is investigating how genome engineering approaches might be used exploit the immune system for new therapies. Much of our research draws on experience from bone marrow and stem cell transplantation, where immune cells & stem cells can be collected, modified and returned to patients. We are particularly interested in harnessing powerful immune effects mediated by engineered T cells that can eradicate some types of blood cancer.  Similar technologies are also being used to repair and rebuild the immune system where there are gaps in immunity leading to infections or auto-immune complications. Through collaborative networks of international researchers, we are working with universities and pharmaceutical/biotechnology companies to develop lentiviral vectors and genome editing technology including TALENS, CRISPR/Cas and base-editing platforms. Clinical trials are underway to investigate the potential of sophisticated engineering of  'universal' genome edited CAR-T cells to eradicate refractory 'hard-to-treat' leukaemia.

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