Gene therapy breakthrough in mice for rare degenerative brain disorder

In a study from the University College London Great Ormond Street Institute of Child Health (UCL GOS ICH) and Great Ormond Street Hospital (GOSH; both London, UK), scientists have for the first time developed a gene therapy targeting dopamine transporter deficiency syndrome (DTDS), a rare degenerative brain disorder. Often mistaken for cerebral palsy, DTDS is a movement disorder that causes dystonia and parkinsonism. It is known to affect around 50 children worldwide, and sadly there is currently no available therapy. In their state-of-the-art study, which will soon progress to clinical trials, the team were able to cure DTDS in mouse...