Zayed Centre for Research team presents gene therapy research at Expo 2020 Dubai

Great Ormond Street paediatric immunologist Claire Booth treats patients with immune system disease

Britain hosted its UK national day at Expo 2020 Dubai on Thursday.

A leading British children’s doctor presented her groundbreaking gene therapy research at Expo 2020 Dubai on Friday, highlighting how modified harmless viruses can be used to treat patients with chronic immune system diseases.

Claire Booth, Mahboubian Professor in Gene Therapy at UCL Great Ormond Institute of Child Health and consultant in paediatric immunology at Great Ormond Street Hospital, delivered a virtual seminar on her world-leading research during the UK pavilion’s “How will we Thrive?” programme.

Great Ormond Street Hospital has been at the cutting edge of the gene therapy field for more than 20 years, with much of its pioneering research now taking place at the Zayed Centre for Research into Rare Disease in Children, which opened in London in 2019.

The centre — a partnership between Great Ormond Street Hospital, the hospital's Children’s Charity and University College London, aims to drive new treatments and cures for children with rare and complex diseases.

“Expo 2020 Dubai is an important global platform for convening discussions about groundbreaking research from around the world. It was a privilege to be able to participate in the UK pavilion’s event and share our progress on 20 years of gene therapy at [Great Ormond Street Hospital],” said Dr Booth.

Britain hosted its UK “national day” at Expo 2020 Dubai on Thursday, featuring diverse music, theatre, arts, poetry, comedy, film, food and innovation across the whole site. It was attended by Britain's Prince William, who made his first official trip to the Emirates this week.

The UK also unveiled a £10 million global tourism campaign on Wednesday as it looks to strengthen closer trade ties with the UAE.

The global initiative to drive inbound tourism to the UK after the sector was hammered by the fallout from the pandemic comes as the country gears up for a year of big events, including Queen Elizabeth II’s platinum jubilee celebrations and the Commonwealth Games in Birmingham.

Claire Booth, consultant in paediatric immunology and gene therapy at Great Ormond Street Hospital, delivered a talk on her work at Expo 2020 Dubai. Photo: Great Ormond Street Hospital

At the “How will we Thrive?” event, Dr Booth outlined how, by using modified harmless viruses, her team were able to correct blood stem cells in patients with immune system diseases.

The approach is also used to treat metabolic diseases and blood disorders, while new gene-editing technologies are also being investigated to improve gene therapy results and treat a wider range of illnesses.

Since opening in 2019, it has hosted more than 105,000 patients and treated more than 25,000 children in Falcon Outpatients facility. A little more than half of the appointments were for children seen for cardiological issues.

Paul Gissen, head of the gene and cell therapy team at the NIHR Great Ormond Street Hospital Biomedical Research Centre, who also works at the Zayed Centre for Research, made a separate presentation at the Genomic Medicine Conference at Expo 2020 Dubai’s Australia pavilion.

Dr Gissen outlined the innovative approaches at Great Ormond Street Hospital for developing therapies for uncurable diseases, highlighting some of his team’s most recent work, including clinical trials for Brineura, an enzyme replacement therapy, for CLN2 Batten Disease, an inherited disorder that primarily affects the nervous system.

He is now exploring using Brineura to save the sight of children affected by the condition.