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GATA2 deficiency and MDS / AML : Experimental strategies for disease modelling and future therapeutic prospects

26 Jun 2022

Abstract excerpt

The importance of predisposition to leukaemia in clinical practice is being increasingly recognized. This is emphasized by the establishment of a novel WHO disease category in 2016 called "myeloid neoplasms with germline predisposition". A major syndrome within this group is GATA2 deficiency, a heterogeneous immunodeficiency syndrome with a very high lifetime risk to develop myelodysplastic syndrome (MDS) and...

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PMID: 35753998
DOI: 10.1111/bjh.18330

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