In the Presidential Symposium, Sofia Gialesaki (Hannover Medical School, Germany) presented her functional studies aimed to understand the pathophysiology behind myeloid leukaemia predisposition in trisomy 21 .
Children with Down syndrome (DS) are at high risk of developing myeloid leukaemia (ML-DS). Up to 30% of DS newborns develop a pre-leukaemic transient abnormal myelopoiesis (TAM), characterised by the accumulation of immature megakaryoblasts of foetal origin. TAM is characterised by GATA1 mutations (GATA1-s) that result in a shorter protein isoform lacking the N-terminal transactivation domain. To understand how trisomy 21 cooperates with GATA1-s in TAM development, the researchers performed a CRISPR/Cas9 screen, targeting the 218 currently annotated coding genes on Hsa21 with 1,090 sgRNAs in both a ML-DS and control cell line.
RUNX1 loss resulted in depletion of ML-DS cells. Additionally, the researchers observed differential RUNX...
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Table of Contents: EHA 2019
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