DUBAI, 11th February, 2022 (WAM) -- As part of the UK Pavilion at Expo 2020 Dubai’s How will we Thrive? week program, Prof Claire Booth, Mahboubian Professor in Gene Therapy at UCL Great Ormond Institute of Child Health (UCL GOS ICH) and Consultant in Paediatric Immunology, Great Ormond Street Hospital (GOSH), was invited to give a virtual seminar to discuss her world-leading research in gene therapy.

GOSH has been at the cutting edge of the gene therapy field for more than 20 years, and much of its pioneering research now takes place at the Zayed Centre for Research into Rare Disease in Children, which opened in London in 2019.

Professor Claire Booth said, "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 GOSH."

Using modified harmless viruses, a team led by Professor Booth and Professor Adrian Thrasher has been able to correct blood stem cells from patients with several immune system diseases, allowing them to develop working immune systems and lead the same lives as their friends. This approach has now been used to treat metabolic diseases and blood disorders, too. New gene-editing technologies are also being investigated to improve gene therapy results and treat a wider range of diseases.

Professor Paul Gissen, Head of the Gene and Cell Therapy Theme at the NIHR GOSH Biomedical Research Centre, is also based at the Zayed Centre for Research and presented at the Genomic Medicine Conference held at Expo 2020 Dubai’s Australia pavilion. Invited by UAE University, Professor Gissen discussed his innovative approaches at GOSH for developing therapies for uncurable diseases, from challenges to opportunities. In some of his team’s most recent work, which he presented at Expo 2020 Dubai, he has been an investigator for the clinical trials for Brineura (an enzyme replacement therapy) for CLN2 Batten Disease, an inherited disorder that primarily affects the nervous system, and is now exploring using Brineura in the eye to save the sight of children with the condition.

The Zayed Centre for Research is a partnership between GOSH, UCL GOS ICH and Great Ormond Street Hospital Children’s Charity, and was made possible thanks to a generous ₤60 million gift in 2014 from HH Sheikha Fatima bint Mubarak, Chairwoman of the General Women's Union, President of the Supreme Council for Motherhood and Childhood, and Supreme Chairwoman of the Family Development Foundation.