Author

Nathan White

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Open access Aug 2026

Functional restoration of immune defects in STAT1 gain-of-function disease following stem cell gene editing.

Efficient and precise correction of STAT1 GOF mutations by base editing is demonstrated, with maintenance of the correction through long-term engraftment in vivo, which represents the first application of gene editing to correct a dominant gain-of-function mutation causing immunodeficiency.

Robert Torrance, K. Orf, Nathan White et al. · Blood · 0 citations