Infants with Artemis-deficient severe combined immunodeficiency improved after corrected genes in their stem cells enabled them to grow their own T cells and B cells, according to a study published in The New England Journal of Medicine.
Mutations in the DNA cross-link repair 1C (DCLRE1C) gene lead to Artemis-deficient severe combined immunodeficiency (ART-SCID), which is characterized by a lack of T lymphocytes with a lack of or nonfunctional B lymphocytes.
ART-SCID responds poorly to typical SCID treatment, allogenic hematopoietic stem cell transplantation (HSCT), even when it involves