[HTML][HTML] A distal single nucleotide polymorphism alters long-range regulation of the PU.1 gene in acute myeloid leukemia

U Steidl, C Steidl, A Ebralidze… - The Journal of …, 2007 - Am Soc Clin Investig
U Steidl, C Steidl, A Ebralidze, B Chapuy, HJ Han, B Will, F Rosenbauer, A Becker…
The Journal of clinical investigation, 2007Am Soc Clin Investig
Targeted disruption of a highly conserved distal enhancer reduces expression of the PU. 1
transcription factor by 80% and leads to acute myeloid leukemia (AML) with frequent
cytogenetic aberrations in mice. Here we identify a SNP within this element in humans that is
more frequent in AML with a complex karyotype, leads to decreased enhancer activity, and
reduces PU. 1 expression in myeloid progenitors in a development-dependent manner. This
SNP inhibits binding of the chromatin-remodeling transcriptional regulator special AT-rich …
Targeted disruption of a highly conserved distal enhancer reduces expression of the PU.1 transcription factor by 80% and leads to acute myeloid leukemia (AML) with frequent cytogenetic aberrations in mice. Here we identify a SNP within this element in humans that is more frequent in AML with a complex karyotype, leads to decreased enhancer activity, and reduces PU.1 expression in myeloid progenitors in a development-dependent manner. This SNP inhibits binding of the chromatin-remodeling transcriptional regulator special AT-rich sequence binding protein 1 (SATB1). Overexpression of SATB1 increased PU.1 expression, and siRNA inhibition of SATB1 downregulated PU.1 expression. Targeted disruption of the distal enhancer led to a loss of regulation of PU.1 by SATB1. Interestingly, disruption of SATB1 in mice led to a selective decrease of PU.1 RNA in specific progenitor types (granulocyte-macrophage and megakaryocyte-erythrocyte progenitors) and a similar effect was observed in AML samples harboring this SNP. Thus we have identified a SNP within a distal enhancer that is associated with a subtype of leukemia and exerts a deleterious effect through remote transcriptional dysregulation in specific progenitor subtypes.
The Journal of Clinical Investigation