Opened seven years ago, the ZCR has become a leading hub for scientific discovery, bringing together scientists, clinicians, and families to accelerate research. Its work spans pioneering gene therapies, genomic diagnoses, regenerative medicine, and innovative treatments.
To mark the anniversary, seven researchers are being spotlighted for their contributions. Professor Manju Kurian focuses on decoding rare neurological diseases, investigating genetic causes and developing precision therapies. Professor Paolo De Coppi, a pioneer in regenerative medicine, works on stem cells, tissue engineering, and innovative surgical techniques for complex conditions.
Dr Amy McTague is finding answers for babies with epilepsy through rapid genome sequencing and stem-cell models. Professor Francesco Muntoni leads research into rare neuromuscular diseases, bridging the gap between genetic discovery and treatment for conditions like Duchenne muscular dystrophy and spinal muscular atrophy.
Professor Waseem Qasim is engineering immune cells to fight cancer, leading pioneering work in genome-edited CAR-T cell therapies. Professor Sara Ghorashian is developing the next generation of immunotherapies for blood cancers, aiming to improve outcomes for children with aggressive forms of the disease.
Professor Claire Booth delivers life-changing gene therapies for children with severe immune disorders, establishing Great Ormond Street Hospital (GOSH) as a global leader in the field. Her research spans laboratory development to clinical studies.
The ZCR, a partnership between Great Ormond Street Hospital, UCL, and Great Ormond Street Hospital Children’s Charity, was made possible by a significant gift from Her Highness Sheikha Fatima bint Mubarak. The centre's mission remains to transform the lives of children with rare and complex diseases through patient-focused research.
