Gene edit success seen
Advanced gene therapy appears to have cured a rare immune disease in all babies tested in recent trials.
US and Canadian scientists say a gene therapy approach has cured 10 infants with a rare disease called Artemis-deficient severe combined immunodeficiency (ART-SCID).
The condition, which causes life-threatening problems with the immune system, affects around one in 65,000 newborns. It is caused by faulty copies of a gene that codes for a DNA repair enzyme called Artemis, which is required for the production of functioning immune cells.
In a recent trial, researchers injected infants with gene-corrected cells after treatment with a chemotherapy drug.
On follow-up, all the infants had genetically corrected and functional immune cells.
The full study is accessible here.