Changes to gene therapy are successful and safer for children with immune disorder

Changes in the way working genes are delivered to children with SCID (Severe Combined Immunodeficiency) could make gene therapy for the disease even safer, finds research led by a team at Great Ormond Street Hospital (GOSH) and its research partner the UCL Institute of Child Health.

Alterations to the delivery method, which are published in the New England Journal of Medicine, leads to a high success rate and the small risk of patients of developing complications, such as cancer, is reduced even further.

SCID is a condition in which children are born without an immune system because of a defect in the gene IL2RG. This condition is sometimes referred to as