Study of Inherited Thrombocytopenia Resulting from Mutations in ETV6 or RUNX1 Using a Human Pluripotent Stem Cell Model

Utilizing CRISPR-Cas9, researchers compared the in vitro phenotype of hematopoietic progenitor cells and megakaryocytes derived from induced pluripotent stem cell lines harboring mutations in either ETV6 or RUNX1.