Therapeutic gene modification in malignant disorders or inherited genetic disorders

Research theme 2

Theme Lead: Professor Amit Nathwani

Gene Therapy

Senior researchers within the BTRU in Stem Cells and Immunotherapies have extensive experience relating to genetic modification of both stem cells and of T-cells in order to correct genetic defects or enhance anti-tumour activity respectively. We used this knowledge and experience to refine strategies to treat inherited genetic disorders by investigating improved vector design and evaluating alternative gene editing technologies - examining their potential to influence the activity of target genes, and, modify T-cell behaviour. Further work focused on advances in chimeric antigen receptor (CAR) T-cell therapies and crossed over with Research Theme 3.