Gene editing holds promise for babies with deadly immune disease

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Gene editing holds promise for babies with deadly immune disease
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Repairs to DNA allow stem cells to develop into T cells.

A T-cell . 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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