
15/07/2025
For the first time in the UK, specialists from the Department of Nuclear Medicine at UCLH - University College London Hospitals NHS Foundation Trust and the Department of Developmental Neurosciences at UCL Institute of Child Health Great Ormond St Hospital London have visualised the effects of cutting-edge gene therapy on the brain using pioneering F-DOPA PET scanning. This provides concrete evidence of changes in neurotransmitter activity, pre and post treatment.
This milestone was part of a life-changing intervention for Gunreet, a toddler with AADC deficiency, treated at GOSH with the newest gene therapy available on the NHS. The scans offer unprecedented insight into how gene therapy alters brain chemistry.
- First NHS-commissioned brain-delivered gene therapy.
- Visualised therapeutic impact using advanced PET imaging.
- Youngest UK patient to receive this treatment.
- Real-world evidence of neurological improvement.
Read the full story: https://buff.ly/WKdbBxe
NHS England
Great Ormond Street Hospital and Charity