Molecular Immunology

Great Ormond Street Hospital (GOSH) and the UCL Institute of Child Health (ICH) have led global advances in treating children born with an inability to fight infections using novel gene therapy approaches.
Children with these conditions, such as severe combined immunodeficiency disease (SCID) have a compromised immune system, which means they are unable to fight infection and have to live in a sterile environment. The conventional treatment for these conditions is bone marrow transplantation, but this does not work for every child.

Professor Adrian Thrasher and Professor Bobby Gaspar are world experts in the field of molecular immunology and novel gene therapy approaches. Their research has transformed the lives of children with these conditions.

Just over 10 years ago they became one of the few teams in the world to begin trials of a new form of therapy for children born with rare genetic disorders. The results from these trials were excellent and to date, fourteen out of sixteen children have been successfully cured.

The team can do even more and hope to make gene therapy available to more children in the future so they too can benefit from the significant clinical improvements. The Centre for Research into Rare Disease in Children will provide the urgently needed space with a dedicated gene therapy facility. This will give them the infrastructure they need to continue to advance and develop this form of genetic medicine for more patients. The work exemplifies the unique partnership between GOSH and the ICH, combining scientific breakthroughs with medicine for patient benefit.

“A decade on, gene therapy has improved the life and health of many children with life threatening immune diseases”