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  • RESEARCH HIGHLIGHT

Gene editing holds promise for babies with deadly immune disease

Repairs to DNA allow stem cells to develop into T cells.
Coloured scanning electron micrograph (SEM) of a T-lymphocyte.

A T-cell (artificially coloured). A rare genetic disease called CD3δ severe combined immune deficiency causes improper development of certain T cells. Credit: David Scharf/SPL

Babies with CD3δ SCID, a rare form of the genetic condition severe combined immune deficiency, have just one abnormal pair of molecules in a single gene, but that’s all it takes to break the immune system. The mutation prevents T cells from detecting pathogens, and most babies with CD3δ SCID die before their second birthday.

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Nature 616, 11 (2023)

doi: https://doi.org/10.1038/d41586-023-00870-z

References

  1. McAuley, G. E. et al. Cell https://doi.org/10.1016/j.cell.2023.02.027 (2023).

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