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Anaïs Marie Møller on Muck Rack

Anaïs Marie Møller

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Articles

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

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By Ryan Wang, Sílvia Puigarnau Coma, Jonas Wolff, Thomas Skov, Didde Haslund, Mette Holm, Martin K. Thomsen, Trine H. Mogensen, Bjarne K. Møller, Rasmus O. Bak, Jacob Giehm Mikkelsen, Sofie R. Dorset, Janni Sjelborg, Sujan Ravendran, Anne Louise S. Revenfeld, Anaïs Marie Møller
| Science Magazine Verified
Abstract Supplementary Materials REFERENCES AND NOTES Information & Authors Metrics & Citations Check Access References Figures Tables Media Share This article has 0 eLetter . Abstract 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.
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