The Zayed Centre for Research, which opened in 2019, focuses on understanding and treating rare diseases in children – and in doing so may offer insights into adult illnesses too.
Linked to the world-renowned Great Ormond Street Hospital for Children (Gosh), it was named in honour of Sheikh Zayed after his wife Sheikha Fatima bint Mubarak donated £60m ($82.6m) to the hospital’s charity in 2014.
As Sheikh Mohamed bin Zayed, Crown Prince of Abu Dhabi and Deputy Supreme Commander of the Armed Forces, visits the centre, we look at its crucial role in combating disease.
Why is the centre needed?
Much work at the Zayed Centre for Research into Rare Disease in Children, to use the full name, relates to conditions with a genetic basis.
Speaking in 2019 to The Lancet Child and Adolescent Health, Prof David Goldblatt, Gosh director of clinical research and development, said most rare diseases with a genetic cause develop in childhood.
There is “a strong imperative to study and treat these diseases” because three in 10 children with a rare condition do not live beyond the age of 5.
While the focus is on rare illnesses, insights in molecular biology and genetics can have wider applications, including for common diseases affecting adults.
The centre is also important in clinical care, having 21 child-friendly consultation rooms and eight clinical investigation rooms.
What does the research cover?
One area is gene therapy, under which healthy forms of genes are inserted into cells to make up for the patient’s “faulty” copies.
It may also involve adding a new gene to produce a protein to combat disease, or disabling a faulty gene.
While still experimental, gene therapy sometimes offers the potential for a permanent cure.
Research also covers cell therapy, where healthy cells are introduced to compensate for diseased ones in the patient.
Another cutting-edge field is regenerative medicine, in which diseased cells, tissues or organs can be repaired, regrown or replaced.
Central to this are therapeutic stem cells, which are “master cells” that have not undergone specialisation into particular types.
The centre has played a part in the first human challenge trial for Covid-19, in which healthy people aged 18 to 30 are being intentionally infected with the coronavirus to help vaccine and treatment development.
What are the research facilities?
The centre, which was purpose built adjacent to Gosh and the University College London Great Ormond Street Institute of Child Health, has world-class facilities.
These include seven rooms where gene and cell therapies can be produced, described as the largest such unit in the UK.
There are 140 laboratory benches “for early development work to test the safety and efficacy of potential new treatments”, nine tissue-culture rooms where new treatments can be tested on lab-grown cells and a flow cytometry suite for the sorting and identification of cells.
At a cardiac research suite, 3-D models of the heart can be produced, which allows treatments and devices to be tailored to the patient.
There are offices for 400 researchers and clinicians, along with meeting areas where teams can collaborate.