Gene therapy cures rare immune disease in all 10 babies tested

Embargoed until: Publicly released: 2022-12-22 09:00

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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's 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. The team injected the infants with gene-corrected cells after treatment with a chemotherapy drug. On follow-up, all the infants had genetically corrected and functional immune cells.

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Journal/
conference: New England Journal of Medicine

Research:Paper

Organisation/s: Smith Cardiovascular Research Institute, USA

Funder: Funded by the California Institute for Regenerative Medicine and the National Institute of Allergy and Infectious Diseases; ClinicalTrials.gov number, NCT03538899.

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