https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/about-our-biomedical-research-centre/our-research-themes/gene-stem-and-cellular-therapies/
Gene, stem and cellular therapies
Theme leads: Professor Paul Gissen
Deputy leads: Professor Claire Booth and Professor David Long
Junior Faculty Representatives: Dr Alice Giustacchini and Dr Nandaki Keshavan
The gene, stem and cellular therapies Theme has pioneered the clinical development of treatments including novel, first-in-child approaches (TALEN and CRISPR-mediated gene-editing, CAR T-cell therapy, new adeno-associated virus gene therapy). Initially delivered by haematopoietic stem cells, new gene therapies are being developed for eye, neurological, neuromuscular, metabolic, bone, skin diseases and solid tumours. Principal investigators in the team have delivered 15 GOSH-manufactured gene and cell products and led 20 early phase gene or cell therapy trials in 2019-2020, with over 10 new trials in set-up in 2022.