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

Scientists and doctors at University College London 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).

source https://medicalxpress.com/news/2021-06-gene-therapy-breakthrough-children-rare.html