Prof Claire Booth

Professor in Gene Therapy and Paediatric Immunology and Consultant Paediatric Immunologist


Professor Claire Booth worked at GOSH since 2005. Professor Booth is a Consultant Paediatric Immunologist at GOSH which is a global centre of excellence and one of only two units commissioned for stem cell transplantation for primary immunodeficiency in the UK.

She is an Academic Clinical Lecturer at University College London (UCL) and a Professor in Gene Therapy and Paediatric Immunology and a Senior Clinical Lecturer in Gene Therapy at UCL Great Ormond Street Institute of Child Health. She also holds a position as Visiting Lecturer, Boston Children’s Hospital/ Dana Farber Cancer Institute (Paediatric Hematology/Oncology).

Professor Claire Booth is a clinical academic, which means she spends a lot of her time undertaking research as well as providing clinical care for inpatients and outpatients.  She undertook her PhD at UCL developing gene therapies for rare immune deficiencies and now continues her research in this field alongside running clinical trials of gene therapy treatments here at GOSH.

She is working in the field of primary immunodeficiency (PID) and gene therapy, leading gene therapy clinical trials at GOSH treating patients with immune deficiencies, haematological and metabolic disorders. She possesses a skill set encompassing pre-clinical laboratory research, translational studies, Phase I/II clinical trials and clinical management of patients with PID, including hematopoietic stem cell transplantation. She is currently Principal Investigator of Phase I/II lentiviral clinical trials of haematopoietic stem cell gene therapy for ADA-SCID, X-SCID and Fanconi Anaemia and Co-Investigator on numerous others. One of her clinical interests is haemophagocytic lymphohistiocytosis (HLH) and she is focused on improving outcomes for HLH patients by developing new therapeutic strategies, including gene therapies.

Professor Booth owns pre-clinical research focuses on the development of gene therapy strategies to treat X-linked lymphoproliferative diseases (XLP-1 and XIAP deficiency) using a conventional lentiviral mediated corrective strategy alongside exploration of novel gene-editing techniques to facilitate targeted gene addition in both haematopoietic stem cells and T cells. 


  • Primary immunodeficiency (PID) 
  • Gene therapy  
  • Hematopoietic stem cell transplantation 
  • Primary immunodeficiency (PID) 
  • Gene therapy  
  • Hematopoietic stem cell transplantation 
  • MBBS 
  • MSc 
  • PhD

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