Gene therapy breakthrough offers hope to children with rare and fatal brain disease

(University College London) Scientists and doctors at UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) and Great Ormond Street Hospital (GOSH) have given hope of a gene therapy cure to children with a rare degenerative brain disorder called Dopamine Transporter Deficiency Syndrome (DTDS). The team have recreated and cured the disease using state-of-the-art laboratory and mouse models of the disease and will soon apply for a clinical trial of the therapy.