RUNX-1 haploinsufficiency causes a marked deficiency of megakaryocyte-biased hematopoietic progenitor cells

B Estevez, S Borst, D Jarocha… - Blood, The Journal …, 2021 - ashpublications.org
Patients with familial platelet disorder with a predisposition to myeloid malignancy (FPDMM)
harbor germline monoallelic mutations in a key hematopoietic transcription factor, RUNX-1.
Previous studies of FPDMM have focused on megakaryocyte (Mk) differentiation and platelet
production and signaling. However, the effects of RUNX-1 haploinsufficiency on
hematopoietic progenitor cells (HPCs) and subsequent megakaryopoiesis remains
incomplete. We studied induced pluripotent stem cell (iPSC)–derived HPCs (iHPCs) and …

RUNX1 haploinsufficiency causes a marked deficiency of megakaryocyte-biased hematopoietic progenitor cells: Mechanistic studies and drug correction

B Estevez, S Borst, D Jarocha, V Sudunagunta… - bioRxiv, 2020 - biorxiv.org
Patients with familial platelet disorder with a predisposition to myeloid malignancy (FPDMM)
harbor germline monoallelic mutations in a key hematopoietic transcription factor RUNX1.
Previous studies of FPDMM have focused on megakaryocyte (Mk) differentiation, and
platelet production and signaling. However, the effects of RUNX1 haploinsufficiency on
hematopoietic progenitor cells (HPCs) and subsequent megakaryopoiesis remains
incomplete. To address this issue, we studied induced-pluripotent stem cell (iPSC)-derived …
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