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T. W. Skov

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#gene editing Sep 2026

Prime editing of human hematopoietic stem cells for correction of GATA2 deficiency.

GATA2 (GATA binding protein 2) deficiency is a severe immunodeficiency caused by heterozygous variants in the gene encoding the transcription factor GATA2. Ex vivo gene editing of a patient's own CD34+ hematopoietic stem and progenitor cells (HSPCs) could provide curative treatment. However, current methods that rely o...

J. H. Wolff, T. W. Skov, S. R. Dorset et al. · 0 citations

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