A mouse model for the basal transcription/DNA repair syndrome trichothiodystrophy

Mol Cell. 1998 Jun;1(7):981-90. doi: 10.1016/s1097-2765(00)80098-2.

Abstract

The sun-sensitive form of the severe neurodevelopmental, brittle hair disorder trichothiodystrophy (TTD) is caused by point mutations in the essential XPB and XPD helicase subunits of the dual functional DNA repair/basal transcription factor TFIIH. The phenotype is hypothesized to be in part derived from a nucleotide excision repair defect and in part from a subtle basal transcription deficiency accounting for the nonrepair TTD features. Using a novel gene-targeting strategy, we have mimicked the causative XPD point mutation of a TTD patient in the mouse. TTD mice reflect to a remarkable extent the human disorder, including brittle hair, developmental abnormalities, reduced life span, UV sensitivity, and skin abnormalities. The cutaneous symptoms are associated with reduced transcription of a skin-specific gene strongly supporting the concept of TTD as a human disease due to inborn defects in basal transcription and DNA repair.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Artificial Gene Fusion
  • Cells, Cultured
  • DNA Helicases*
  • DNA Repair / genetics*
  • DNA-Binding Proteins*
  • Disease Models, Animal
  • Female
  • Growth / genetics
  • Growth / physiology
  • Hair / abnormalities
  • Hair Diseases / genetics*
  • Hair Diseases / mortality
  • Hair Diseases / physiopathology
  • Humans
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Mutant Strains
  • Mutagenesis, Site-Directed
  • Mutation / genetics
  • Proteins / genetics
  • Proteins / physiology
  • Skin / metabolism
  • Skin / pathology
  • Survival Analysis
  • Syndrome
  • Transcription Factors / genetics
  • Transcription Factors / physiology
  • Transcription, Genetic / genetics
  • Xeroderma Pigmentosum / genetics
  • Xeroderma Pigmentosum Group D Protein

Substances

  • DNA-Binding Proteins
  • Proteins
  • Transcription Factors
  • DNA Helicases
  • Xeroderma Pigmentosum Group D Protein
  • ERCC2 protein, human
  • Ercc2 protein, mouse