Development of new gene therapies and assays for SCID
Research type
Research Study
Full title
Development of new gene therapies and assays for severe combined immunodeficiency (SCID)
IRAS ID
356890
Contact name
Claire Booth
Contact email
Sponsor organisation
UCL-Great Ormond Street Institute of Child Health
Clinicaltrials.gov Identifier
22IC11, Additional R&D; 24IC02, Additional R&D
Duration of Study in the UK
4 years, 11 months, 18 days
Research summary
Severe combined immunodeficiency (SCID) is a group of life-threatening inherited disorders resulting in severely impaired immune system development and function. Gene therapy is an attractive curative treatment strategy, as it avoids the need for bone marrow transplant and could improve patient outcomes. Clinical trials performed here at Great Ormond Street Hospital (GOSH) have already demonstrated the long-term clinical efficacy and safety profiles of gene therapies for some forms of SCID. We are now aiming to increase the scope of our research in the development of new gene therapies for SCID. As well as expanding on current methods, including viral-mediated gene addition, we will also explore novel gene editing techniques and in vivo strategies. We also aim to develop new assays which are essential for the screening, diagnosis and monitoring of SCID. Additionally, improving the understanding of disease mechanisms underlying the clinical features of SCID is essential in targeting new therapies. Patient tissue, including blood cells, bone marrow stem cells and skin biopsies, is essential for these study goals. We plan to use these patient cells in vitro to test these new therapies and assays. We may also want to test corrected patient cells in an in vivo mouse model of the disease, which is necessary before gene therapy treatments can move to clinical trial.
REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
25/EE/0184
Date of REC Opinion
23 Sep 2025
REC opinion
Further Information Favourable Opinion