News and features

In a world first, researchers at UCL Great Ormond Street Institute of Child Health have grown ‘mini-eyes' to better understand the development of blindness in a rare genetic disease called Usher syndrome.

In a world first, researchers at GOSH and the UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) have used genetically modified cells – created using a new technique called base editing – to treat a patient with previously incurable leu…

This Childhood Cancer Awareness Month, we share the news that Great Ormond Street Hospital Children’s Charity (GOSH Charity) is funding a pioneering clinical trial at GOSH. This trial provides hope for treatment for a notoriously difficult to treat …

The first child to be treated in the UK with gene therapy celebrates his 21st birthday this week, twenty years after having the pioneering treatment at Great Ormond Street Hospital for Children (GOSH).