Researchers and children have produced a short animation explaining gene therapy.
The animation aimed at both children and adults was developed by the NIHR Blood and Transplant Research Unit in Stem Cells and Immunotherapies at UCL (UCL BTRU) – which is led by UCLH clinicians Dr Claire Roddie and Professor Karl Peggs – together with members of the Young Persons’ Advisory Group (YPAG) at Great Ormond Street Hospital for Children.
The animation explains how missing or faulty genes can cause illness, and how inserting working copies of genes into our stem cells – cells found in bone marrow which develop into all the different kinds of cells – can help treat these illnesses.
In the animation, Alexis and Freddie, two members of the Young Persons’ Advisory Group at GOSH, ask questions about gene therapy. Katie Snell, Lead Gene Therapy Research Nurse at UCL Great Ormond Street Institute of Child Health, answers these questions.
Professor Adrian Thrasher, a Research Theme Lead for the UCL BTRU, said: “I spent most of my career as a researcher developing gene therapies for children who have an immune system that doesn’t function properly. The immune system of these children can’t protect them from infections, and they can become life-threatening.
“A lot is said on the news about gene editing, less how it can help to treat inherited diseases. Alexis and Freddie helped us to explain this brilliantly in our animation. We hope it finds much interest and explains a ground-breaking future treatment for some inherited conditions.”