[HTML][HTML] Targeted gene correction of RUNX1 in induced pluripotent stem cells derived from familial platelet disorder with propensity to myeloid malignancy restores …
Highlights•Induced pluripotent stem cells were generated from dermal fibroblasts of a patient
with familial platelet disorder (FPD) with propensity to myeloid malignancy.•The induced
pluripotent stem cells exhibited a prominent dysfunction in megakaryocytic differentiation.•
Transcription activator-like effector nuclease-mediated correction of the RUNX1 mutation
restored megakaryopoiesis in the induced pluripotent stem cells.Familial platelet disorder
with propensity to acute myeloid leukemia (FPD/AML) is an autosomal dominant disease …
with familial platelet disorder (FPD) with propensity to myeloid malignancy.•The induced
pluripotent stem cells exhibited a prominent dysfunction in megakaryocytic differentiation.•
Transcription activator-like effector nuclease-mediated correction of the RUNX1 mutation
restored megakaryopoiesis in the induced pluripotent stem cells.Familial platelet disorder
with propensity to acute myeloid leukemia (FPD/AML) is an autosomal dominant disease …
