An international team led by researchers at GOSH, UCL and the NIHR GOSH BRC have developed in mice a gene therapy that significantly reduces the hearing loss associated with Norrie disease.
An international research collaboration, including GOSH and our research partner UCL Institute of Child Health, has shown that a technique known as rapid genome sequencing can provide a diagnosis for 43 per cent of children with unexplained epilepsy.
Researchers from across GOSH, UCL GOS ICH and the Wellcome Sanger Institute have published new insights that explain why some children have a longer remission than others after having cutting-edge CAR T-cell therapy for leukaemia.