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

Alina Shrourou19 January 2021

Written by Linda von Nerée, NIHR Blood and Transplant Research Unit in Stem Cells and Immunotherapies at UCL.

How many pairs of jeans do you have in your wardrobe? How many genes are in your body? What are genes anyway and do you know how they can help to treat an illness?!

All is explained in this brand-new animation from us at the NIHR Blood and Transplant Research Unit in Stem Cells and Immunotherapies at University College London (UCL BTRU). Well, except how many jeans you own, that stays your secret.

Animated children asking questions about gene therapy

Young people asking questions about gene therapy in the animation ‘Gene Therapy explained: Changing our bodies’ recipe to treat disease’. Screenshot from an animation provided by KindeaLabs.

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 those that affect the ability of our blood’s immune system to fight off infections that make us ill.

The animation shows, Alexis and Freddie, two members of the Young Persons’ Advisory Group (YPAG) at Great Ormond Street Hospital for Children asking questions to understand what gene therapy is about. All members of the group were involved in shaping the animation and they regularly work with doctors, nurses and scientists helping to improve health care research for children. When possible, the group meets near the Zayed Centre for Research into Rare Disease in Children, where scientists look for new and better ways to treat uncommon diseases in children.

Why this Gene Therapy 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 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 Gene Therapy 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

‘It was a huge pleasure to work with Alexis, Freddie and YPAG as a group of inspiring young people involved in improving health through research. Their ideas and invaluable input made the animation so much better and very different from the first draft we presented back to them at a meeting in summer 2018.’ – Linda von Nerée, Patient and Public Involvement Lead at NIHR Blood and Transplant Research Unit in Stem Cells and Immunotherapies at UCL

‘It is such a privilege to work in the gene therapy field and see research in action. I had a great time attending the YPAG group and hearing from its members. Alexis and Freddie have done a great job!’ Katie Snell, Lead Gene Therapy Research Nurse at UCL Great Ormond Street Institute of Child Health

Young Persons’ Advisory Group (YPAG) at Great Ormond Street Hospital for Children

The Young Persons’ Advisory Group (YPAG) at Great Ormond Street Hospital for Children – young people making health care research for children better

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.

Learn more in the full animation:

‘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!

About the authors

  • Alexis – I joined YPAG when I was 8 years old and I have been a member for 5 years! Including the voices of young people is important because we are the next up and coming generation, and in a few years we will be the ones filling these roles so I think it’s important we have a say in how our future is going to be like.
  • Freddie – I am 12 years old and I joined YPAG when I was 9. I really enjoy YPAG because I learn something new every time and get to be involved in interesting things like this animation.
  • Linda – In my role, I bring together patients, members of the public, researchers, doctors and nurses to learn from each other and design research in the best possible way for those to benefit from it. Working with YPAG is a huge pleasure!

About the Young Persons’ Advisory Group (YPAG)

YPAG logo

We are a group of young people working with doctors, nurses and researchers to add our views and opinions to the development of new treatments for children. We are part of GenerationR, a network of young people improving health through research.

More at: https://www.gosh.nhs.uk/research-and-innovation/nihr-gosh-brc/patient-and-public-involvement

About the NIHR Blood and Transplant Research Unit in Stem Cells and Immunotherapies at University College London

The NIHR Blood and Transplant Research Unit (BTRU) in Stem Cell and Immunotherapies at University College London (UCL) is an academic partnership with NHS Blood and Transplant funded by the National Institute for Health Research (NIHR). It focusses on improving stem cell transplants (transfer of stem cells, which lead to new blood cells in the recipient) and the use of novel therapies, including CAR-T and gene therapy, both to treat inherited genetic disorders and to repair or strengthen the immune system’s ability to fight infection or disease. For more information, please visit https://www.ucl.ac.uk/cancer/research/centres-and-networks/nihr-blood-and-transplant-research-unit-stem-cells-and-immunotherapies or follow @BTRUinStemCells on twitter.

Contact for any questions or inquires: Linda von Nerée, Patient and Public Involvement Lead at NIHR Blood and Transplant Research Unit in Stem Cells and Immunotherapies at University College London, email: l.vonneree@ucl.ac.ukNIHR BTRU logo