Severe anemia in the Nan mutant mouse caused by sequence-selective disruption of erythroid Kruppel-like factor.

Document Type

Article

Publication Date

2010

Keywords

Anemia, Animals, Base-Sequence, Binding-Sites, Chromosome-Mapping, DNA, Disease-Models-Animal, Erythrocytes, Gene-Expression, Genes-Reporter, Hemoglobins, Heterozygote, Humans, K562-Cells, Kruppel-Like-Transcription-Factors, Mice-Inbred-C3H, Mice-Mutant-Strains, Models-Molecular, Mutant-Proteins, Mutation-Missense, Phenotype, Pregnancy, Promoter-Regions-Genetic, Transcriptional-Activation, Zinc-Fingers

First Page

15151

Last Page

15156

JAX Source

Proc Natl Acad Sci U S A 2010 Aug; 107(34):15151-6.

Abstract

Studies of mouse models of anemia have long provided fundamental insights into red blood cell formation and function. Here we show that the semidominant mouse mutation Nan ("neonatal anemia") carries a single amino acid change (E339D) within the second zinc finger of the erythroid Kruppel-like factor (EKLF), a critical erythroid regulatory transcription factor. The mutation alters the DNA-binding specificity of EKLF so that it no longer binds promoters of a subset of its DNA targets. Remarkably, even when mutant Nan and wild-type EKLF alleles are expressed at equivalent levels, the mutant form selectively interferes with expression of EKLF target genes whose promoter elements it no longer binds. This interference yields a distorted genetic output and selective protein deficiencies that differ from those seen in EKLF-heterozygous and EKLF-null red blood cells and presents a unique and unexpected mechanism of inherited disease.

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