International Journal of Hematology

DOI: 10.1007/s12185-018-2488-1 Pages: 28-34

Targeting transcription factors in acute myeloid leukemia

1. Beth Israel Deaconess Medical Center and Harvard Medical School, Department of Medicine

2. Gunma University Graduate School of Medicine, Department of Hematology

3. National Cancer Center, Division of Translational Genomics, Exploratory Oncology Research and Clinical Trial Center

4. Harvard Medical School, Harvard Stem Cell Institute

Correspondence to:
Susumu S. Kobayashi
Tel: +81-4-7133-1111



Transcription factors recognize and bind to consensus sequence elements that are specific for each transcription factor, and the transcription factors then regulate downstream gene expression. In the bone marrow, transcription factors, such as C/EBPα, PU.1, and RUNX1, control essential genes to maintain the normal hematopoietic system. Dysregulation of transcription factors caused by gene mutations, chromosomal aberrations, or aberrant expression can lead to cancer, including acute myeloid leukemia. In the past, transcription factors were not considered “druggable” targets. However, a better understanding of the pathology of malignant tumors and mechanisms of transcriptional regulation has enabled us to develop novel therapeutic strategies that target transcription factors. In this review, we focus on transcription factors that play important roles in leukemogenesis and current efforts and prospects in the development of transcriptional therapy. We believe that such a therapeutic approach will benefit patients with cancers that involve acute myeloid leukemia in the near future.

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