LONDON- The Zayed Centre for Research into Rare Disease in Children (ZCR) in London recently celebrated its second anniversary, marking two years of significant scientific achievements and milestones.

Notably, since its opening, the Centre has treated 105,000 young patients, been involved in several globally-significant research projects, and hosted dignitaries from Abu Dhabi, including His Highness Sheikh Mohamed bin Zayed Al Nahyan, Crown Prince of Abu Dhabi and Deputy Supreme Commander of the UAE Armed Forces, who toured ZCR in September and spoke with researchers, professors and doctors to learn more about the Centre’s lifesaving research and treatments.

Since the onset of the pandemic, the Zayed Centre for Research has played an instrumental role in the world’s first human challenge study of COVID-19. The study explores the smallest amount of virus needed to cause infection, giving doctors and scientists greater understanding of COVID-19. In turn, this research has supported the pandemic response by aiding vaccine and treatment development.

July marked 20 years since the first patient was treated with gene therapy at Great Ormond Street Hospital and much of the pioneering gene therapy research at the hospital now takes place at the Zayed Centre for Research. Professor Claire Booth recently celebrated the successful results of a stem cell gene therapy clinical trial for a form of severe combined immunodeficiency called ADA-SCID in a joint study with UCLA. Of the 50 patients treated in the trial, 48 are no longer showing symptoms of the disease.

Professor Manju Kurian, who discovered the brain disorder dopamine transporter deficiency syndrome (DTDS) in 2009, has led development of a potential gene therapy cure for the disease.

Professor Kurian said, "Being in the Zayed Centre has been absolutely life-transforming for both me and my researchers. We answer the questions that families ask us: why has my child got this condition, how has this happened and what can you do to make my child’s life better? And being in the Zayed Centre has allowed us to answer them. We have the most amazing laboratory space, we have state-of-the-art tissue culture facilities and we are in a milieu of other researchers who have the same goal, propelling us forward for these children."GOSH’s credentials as a global leader in gene therapy have also led to a new partnership with ViroCell Biologics, a global expert in vector manufacture. The Zayed Centre for Research will become a central hub for the production of vectors - the gene therapy ‘delivery system’ that takes the working gene into living cells.

In addition, a team from the Abu Dhabi Stem Cells Centre (ADSCC) took part in a workshop at ZCR last November to explore future collaborations in the areas of rare diseases, gene therapy and regenerative medicine.

The ADSCC team included Dr. Hawaa Al Mansoori, Member of the Federal National Council and Deputy Medical Director of Imperial College London Diabetes Center and Executive Director, Division of Intramural Research, ADSCC; Professor Maha Barakat, Director General of the Frontline Heroes Office; Dr Fatima Al Kaabi, Executive Director of the Abu Dhabi-Bone Marrow Transplant program, ADSCC; and Dr Yendry Ventura, General Manager, ADSCC.

On this occasion, Dr. Fatima Al Kaabi said, "Future collaborations with the Zayed Centre for Research will provide us with further opportunities to share research and expertise that can help us treat rare genetic diseases specific to the UAE population." The Zayed Centre for Research is a partnership between Great Ormond Street Hospital (GOSH), University College London and Great Ormond Street Hospital Children’s Charity, and was made possible thanks to a generous ?60 million gift in 2014 from H.H. Sheikha Fatima bint Mubarak, Chairwoman of the General Women's Union (GWU), President of the Supreme Council for Motherhood and Childhood, Supreme Chairwoman of the Family Development Foundation (FDF), and the "Mother of the Nation".

 

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