Scientists and doctors at UCL GOS ICH and GOSH have given hope of a gene therapy cure to children with a rare degenerative brain disorder called Dopamine Transporter Deficiency Syndrome (DTDS)
Scientists studying the effectiveness of CAR T-cell therapies in children with leukaemia have discovered a small sub-set of T-cells that are likely to play a key role in whether the treatment is successful
An international team of researchers at GOSH and UCLA have developed a gene therapy that successfully treated 48 out of 50 children with a form of severe combined immunodeficiency