Cookies on this website

We use cookies to ensure that we give you the best experience on our website. If you click 'Accept all cookies' we'll assume that you are happy to receive all cookies and you won't see this message again. If you click 'Reject all non-essential cookies' only necessary cookies providing core functionality such as security, network management, and accessibility will be enabled. Click 'Find out more' for information on how to change your cookie settings.

Mutations in the CEBPA gene are present in 7%-10% of human patients with acute myeloid leukemia (AML). However, no genetic models exist that demonstrate their etiological relevance. To mimic the most common mutations affecting CEBPA-that is, those leading to loss of the 42 kDa C/EBPalpha isoform (p42) while retaining the 30kDa isoform (p30)-we modified the mouse Cebpa locus to express only p30. p30 supported the formation of granulocyte-macrophage progenitors. However, p42 was required for control of myeloid progenitor proliferation, and p42-deficient mice developed AML with complete penetrance. p42-deficient leukemia could be transferred by a Mac1+c-Kit+ population that gave rise only to myeloid cells in recipient mice. Expression profiling of this population against normal Mac1+c-Kit+ progenitors revealed a signature shared with MLL-AF9-transformed AML.

Original publication




Journal article


Cancer Cell

Publication Date





299 - 310


Animals, CCAAT-Enhancer-Binding Protein-alpha, Cell Differentiation, Disease Progression, Gene Expression Profiling, Gene Expression Regulation, Leukemic, Granulocytes, Leukemia, Myelomonocytic, Acute, Macrophage-1 Antigen, Mice, Mice, Knockout, Models, Biological, Mutant Proteins, Myeloid Progenitor Cells, Neoplasm Transplantation, Neoplastic Stem Cells, Phenotype, Protein Isoforms, Proto-Oncogene Proteins c-kit