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Sharing the science of gene therapy

by Guest Author on 9 Jan 2019

This festive season, stem cell scientist Professor Bobby Gaspar, from the UCL Great Ormond Street Institute of Child Health, appeared as a special guest on the BBC Royal Institution Christmas Lectures. Here he shares the thrill of healing patients using gene therapy – and why it’s so important to communicate the science behind new medicines to the world.

Professor Aoife McLysaght, gene therapy patient Rhys & Professor Bobby Gaspar. Image: Paul Wilkinson Photography

Professor Aoife McLysaght, gene therapy patient Rhys & Professor Bobby Gaspar. Image: Paul Wilkinson Photography

To be a part of the Christmas Lectures alongside Rhys, the first patient to be successfully treated at Great Ormond Street Hospital with gene therapy back in 2001, was very special.

I first became fascinated by the body and how it works when I was a teenager. This led to me joining Great Ormond Street Hospital as a physician 25 years ago. I was immediately struck by the clear need to find a better treatment for children suffering from severe combined immunodeficiency diseases. These conditions leave children extremely vulnerable to infectious diseases from birth and can be fatal.

A futuristic concept

At the time, the idea of gene therapy sounded like science-fiction to me. But I was intrigued and motivated to investigate whether the use of a genetic therapy for these diseases could lead to an effective treatment.

We successfully treated our first patient in 2012 and we’ve treated another 29 patients since using their own blood stem cells. The cells are genetically corrected outside of the body by replacing the missing or faulty gene, then transplanted back into the body. Using the patient’s own stem cells from their bone marrow provides a perfect match and eliminates the need for a donor search, along with the associated risk of complications such as transplant rejection.

Getting to this point has been a massive team effort – it really does take a whole village. Expert input and fresh ideas from people specialising in a vast range of disciplines have been vital. It’s involved scientists doing bench-based lab work investigating the nature of cells and the genes within them, laboratory staff who engineer the patient cells, doctors and nurses who treat and look after our patients, as well as the patients themselves and their families.

Communicating our research

I think it’s important to devote time and effort to figure out how to best communicate research in an accessible way. Not everyone needs, or wants, to know the full intricacies of the research. But they might still be interested in the puzzle we’re trying to understand and why finding the answer could make a difference to people’s lives.

Gene therapy can be an emotive issue for many people. I’ve heard genes being referred to as the ‘stuff of our souls’. This means it’s an area of research which can so easily lead to controversy or contention if the aims and actions of scientists are misunderstood. This makes it all the more important to help people understand what we’re doing with the technology.

Medicine, doctors and scientists of the future

The way I describe gene therapy is as a potentially new form of medicine, made up of genetic material. We want to use it to eliminate a disease, not change a person or their soul. Added to this, the gene-corrected cells cannot be passed on to the next generation.

Engaging with young audiences like that of the Lectures is so important because they are our society of the future, they are where the next generation of scientists and physicians will come from. If just one young person watching the programme is inspired to pursue medical research or become a physician as a result, then I’ll be happy.

Prof Adrian Thrasher has been Bobby’s co-investigator throughout his MRC-funded severe combined immunodeficiency diseases research.

Watch the lecture Who Am I? : What Makes Me, Me? on BBC iPlayer



Superb read of brilliant applied science.
Profound thanks to all involved.

author avatar by David Robertson on 09-Jan-2019 16:49

How do I get a stem cell therapy done for my son of 8 years d born with right side hempligia (cerebal pawsy) please help me to help my son he’s the medical mirical he walks rams and against the odds 1/3rd brain damaged via stroke just as he was born lack of oxeygen email

author avatar by Sarah on 10-Jan-2019 00:53

Genetic Engineering is a set of technologies used to change the genetic makeup of cells, including the transfer of genes within and across species boundaries to produce improved or novel organisms. It will help to cure many genetic issues in future. It has high scope to build career in it. Thanks for informative article, keep sharing.

author avatar by Genetic Engineering on 15-Jan-2019 07:32

Wow. Never heard of Gene therapy, you made me intrigued

author avatar by Body Rubs Kansas City on 07-Feb-2019 06:08

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