Zayed Centre for Research powers ahead with seven experts at the helm
Most complex surgery involves operations on a single patient but there are procedures that involve two. That is work in vitro such as that carried out by Prof Paolo De Coppi, who works at London's Zayed Centre for Research into Rare Disease in Children, the dedicated facility that pursues genetic and regenerative breakthroughs for conditions affecting children. Precision implants Prof De Coppi…
The Zayed Centre for Research (ZCR) has made significant strides in medical research, spearheaded by a team of seven leading experts. Prof Paolo De Coppi, a prominent figure at the centre, focuses on the development of mini-organs to replace organs in children suffering from serious medical conditions. These "mini-organs" are developed in collaboration with various specialists, including stem-cell biologists, gene-sequence experts, and bioengineers.
One of their most innovative techniques is spatial transcriptomics, which examines gene expression in three dimensions, providing valuable insights into cell behavior within a tissue context.
The Zayed Centre, which opened in 2019, is an interdisciplinary hub for researchers and clinicians under one roof, partnered with the prestigious Great Ormond Street Hospital. It aims to develop gene and cell therapies for seriously ill children, both in the UK and internationally. The centre's entrance invites families and patients to observe the ongoing research and engage with the scientific community, fostering transparency and collaboration.
Prof Manju Kurian, a consultant paediatric neurologist at ZCR, leads a team that seeks to identify genetic causes of childhood neurological disorders and develop precision therapies, including novel gene therapies. Dr Amy McTague focuses on early onset epilepsy, using genomics to enhance diagnosis and treatment outcomes. Prof Francesco Muntoni, a neuromuscular disease specialist, bridges the gap between genetic discovery and treatment, contributing to groundbreaking therapies for conditions like Duchenne muscular dystrophy and spinal muscular atrophy.
Prof Waseem Qasim leads research on cell and gene therapy, focusing on immune cells to treat children with cancer and immune disorders. His pioneering work in genome-edited CAR-T cell therapies has led to world-first treatments for children with untreatable leukemia.
Sara Ghorashian, a consultant paediatric haematologist, conducts research on CAR-T cell therapies and cellular immunotherapy, aiming to improve outcomes for high-risk or relapsed leukemia patients. Prof Claire Booth, a paediatric immunologist, leads multiple gene therapy clinical trials, developing treatments for children with rare immune deficiencies and other inherited disorders.
Since its opening seven years ago, the Zayed Centre has provided expert care to over 13,300 children with rare and complex conditions, thanks to a £60 million gift from Sheikha Fatima bint Mubarak.
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