A noncoding point mutation of Zeb1 causes multiple developmental malformations and obesity in Twirler mice

PLoS Genet. 2011 Sep;7(9):e1002307. doi: 10.1371/journal.pgen.1002307. Epub 2011 Sep 29.

Abstract

Heterozygous Twirler (Tw) mice develop obesity and circling behavior associated with malformations of the inner ear, whereas homozygous Tw mice have cleft palate and die shortly after birth. Zeb1 is a zinc finger protein that contributes to mesenchymal cell fate by repression of genes whose expression defines epithelial cell identity. This developmental pathway is disrupted in inner ears of Tw/Tw mice. The purpose of our study was to comprehensively characterize the Twirler phenotype and to identify the causative mutation. The Tw/+ inner ear phenotype includes irregularities of the semicircular canals, abnormal utricular otoconia, a shortened cochlear duct, and hearing loss, whereas Tw/Tw ears are severely malformed with barely recognizable anatomy. Tw/+ mice have obesity associated with insulin-resistance and have lymphoid organ hypoplasia. We identified a noncoding nucleotide substitution, c.58+181G>A, in the first intron of the Tw allele of Zeb1 (Zeb1(Tw)). A knockin mouse model of c.58+181G>A recapitulated the Tw phenotype, whereas a wild-type knockin control did not, confirming the mutation as pathogenic. c.58+181G>A does not affect splicing but disrupts a predicted site for Myb protein binding, which we confirmed in vitro. In comparison, homozygosity for a targeted deletion of exon 1 of mouse Zeb1, Zeb1(ΔEx1), is associated with a subtle abnormality of the lateral semicircular canal that is different than those in Tw mice. Expression analyses of E13.5 Twirler and Zeb1(ΔEx1) ears confirm that Zeb1(ΔEx1) is a null allele, whereas Zeb1(Tw) RNA is expressed at increased levels in comparison to wild-type Zeb1. We conclude that a noncoding point mutation of Zeb1 acts via a gain-of-function to disrupt regulation of Zeb1(Tw) expression, epithelial-mesenchymal cell fate or interactions, and structural development of the inner ear in Twirler mice. This is a novel mechanism underlying disorders of hearing or balance.

Publication types

  • Research Support, N.I.H., Intramural
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Abnormalities, Multiple / genetics*
  • Animals
  • Binding Sites / genetics
  • Carrier Proteins / genetics
  • Chromosome Mapping
  • DNA-Binding Proteins / genetics
  • Ear, Inner / abnormalities*
  • Epithelial-Mesenchymal Transition / genetics
  • Gene Expression Regulation, Developmental
  • Gene Knock-In Techniques
  • Homeodomain Proteins / genetics*
  • Homeodomain Proteins / metabolism*
  • Introns / genetics*
  • Kruppel-Like Transcription Factors / genetics*
  • Kruppel-Like Transcription Factors / metabolism*
  • Mice
  • Mice, Inbred C57BL
  • Nuclear Proteins / genetics
  • Obesity / genetics*
  • Phenotype
  • Point Mutation / genetics
  • RNA, Untranslated / genetics
  • RNA-Binding Proteins
  • Transcription Factors
  • Zinc Finger E-box-Binding Homeobox 1

Substances

  • Carrier Proteins
  • DNA-Binding Proteins
  • Homeodomain Proteins
  • Kruppel-Like Transcription Factors
  • Mybbp1a protein, mouse
  • Nuclear Proteins
  • RNA, Untranslated
  • RNA-Binding Proteins
  • Transcription Factors
  • ZEB1 protein, mouse
  • Zinc Finger E-box-Binding Homeobox 1