‘Gene Therapy explained: Changing our bodies’ recipe to treat disease’

By London YPAG,



Guess how many pairs of jeans you have in your wardrobe! Now, guess how many genes are in your body. Are you wondering what genes are anyway and how they can help to treat an illness?
We worked on a brand-new animation to explain exactly that. Well, except how many jeans each of us owns, that stays our & your secret.

A very new treatment, called ‘gene therapy’ helps to treat some inherited diseases passed on from parent to child that don’t have a treatment or cure yet. Many different gene therapies are currently in development all over the world for many inherited diseases, such as some hiccups in our blood’s immune system which can’t fight off infections that make us ill.

All members of our group were involved in shaping the animation. We, Alexis and Freddie, are featured in the animation asking questions to understand what gene therapy is all about.

Why this animation?

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 brilliantly explain just this in our animation. We hope it finds much interest and explains a ground-breaking future treatment for some inherited conditions.’ – Adrian Thrasher, Professor in Paediatric Immunology and Research Lead at the NIHR Blood and Transplant Research Unit in Stem Cells and Immunotherapies at University College London (UCL)

What was it like to work on the animation?

It is a new and innovative way to treat some inherited diseases, which surprised me because I thought there were a few other remedies and cures already out there. I really like the animation, and I’m so glad it has turned out this well, (especially the hair), I am so grateful to have had an opportunity to be a part of this!Alexis, member of the Young Persons’ Advisory Group (YPAG) at Great Ormond Street Hospital for Children

‘I enjoyed being part of the animation because I have never done anything like that before. Because of the lockdown I went in my bedroom and recorded my voice on a phone which was strange, but I think the finished animation is good.’Freddie, YPAG member at Great Ormond Street Hospital for Children

Did you know?

Researchers estimate that we have between 20,000 and 25,000 genes in our body. We have two copies of each gene, one from each parent.

Now, we present you with the result of our work: ‘Gene Therapy explained: Changing our bodies’ recipe to treat disease’
Let us know what you think and if you like it.  Please share widely with your friends and family!