Genes to cells,
& cells to patients
At Great Ormond Street Institute of Child Health, our group is investigating new treatments for inherited diseases and approaches to use the immune system to treat certain types of cancer. Most of our projects draw on experience from bone marrow and stem cell transplantation, where cells can be harvested and manipulated in special clean room laboratories. We are particularly interested in harnessing powerful effects mediated by T cells, but are also using the same technologies for other conditions, including keratinocytes for skin disorders. Through collaborative networks of international researchers, we are working with pharmaceutical companies and biotechnology firms to advance next-generation molecular tools, including TALENs and CRISPR/Cas9 for targeted gene editing. Early applications have already included 'universal' T cells where lentiviral delivery of chimeric antigen receptors has been combined with 'genome-editing' to provide new leukaemia treatments. Next generation cell therapies are being developed using base-editing technology for childhood diseases.