Article
iPSC modeling of stage-specific leukemogenesis reveals BAALC as a key oncogene in severe congenital neutropenia.
Cell stem cell - 6 May 2021
Dannenmann Benjamin, Klimiankou Maksim, Oswald Benedikt, Solovyeva Anna, Mardan Jehan, Nasri Masoud, Ritter Malte, Zahabi Azadeh, Arreba-Tutusaus Patricia, Mir Perihan, Stein Frederic, Kandabarau Siarhei, Lachmann Nico, Moritz Thomas, Morishima Tatsuya, Konantz Martina, Lengerke Claudia, Ripperger Tim, Steinemann Doris, Erlacher Miriam, Niemeyer Charlotte M, Zeidler Cornelia, Welte Karl, Skokowa Julia
Abstract excerpt
Severe congenital neutropenia (CN) is a pre-leukemic bone marrow failure syndrome that can evolve to acute myeloid leukemia (AML). Mutations in CSF3R and RUNX1 are frequently observed in CN patients, although how they drive the transition from CN to AML (CN/AML) is unclear. Here we establish a model of stepwise leukemogenesis in CN/AML using CRISPR-Cas9 gene editing of CN patient-derived iPSCs. We identified...
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