Refinement of gene and cell therapies for inherited immunodeficiencies based on human interventions and developing technologies


  • Prof Adrian Thrasher

    University College London

Project summary

Adrian is Professor of Paediatric Immunology at the Institute of Child Health, University College London, and Honorary Consultant Paediatric Immunologist at Great Ormond Street Hospital for Children NHS Foundation Trust. His current PRF programme is focused on the following themes: development of human stem cell (HSC) gene therapy for primary immunodeficiencies (PID) as a medical need; utilising PIDs as a paradigm for HSC gene therapy; identification of mechanisms leading to toxicities; redesign of and pre-clinical evaluation of transgene additive vectors; development of refined technologies to target genetic modifications; and evaluation of reduced-intensity conditioning strategies for engraftment of gene-corrected cells.