Different genes, different diabetes: lessons from maturity-onset diabetes of the young

Ann Med. 2002;34(3):207-16.

Abstract

Maturity-onset diabetes of the young (MODY) is a genetic subgroup of diabetes characterised by an autosomal dominant inheritance and early onset, non-insulin dependent diabetes. This results from a monogenic defect causing beta-cell dysfunction. The defining of five genes in which mutations cause MODY has allowed us to understand the clinical heterogeneity seen in this condition and can guide clinical management. Mutations in the glucokinase gene lead to stable hyperglycaemia, complications are unusual and treatment is rarely needed. Glucokinase patients are often detected during screening in pregnancy. While maternal mutations increase birth weight by increasing maternal glycaemia, fetal mutations reduce birth weight by reducing fetal insulin secretion. Patients with mutation in genes encoding the transcription factors, hepatocyte nuclear factor (HNF)- 1alpha, HNF-4alpha, HNF-1beta and insulin promoter factor 1 (IPF-1) have a common progressive beta-cell failure resulting in increasing hyperglycaemia and treatment requirements. These patients are at risk of developing microvascular complications. They show a pharmacogenetic effect with a specific sensitivity to sulphonylureas. Patients with transcription factor mutations have a range of discrete extra-pancreatic phenotypes including a low renal threshold for glucose with HNF-1alpha mutations, altered lipids and lipoproteins with HNF-4alpha mutations and a variety of cystic renal diseases and uterine and genital developmental disorders with HNF-1beta mutations. Molecular genetic testing is now available in routine clinical practice. This allows confirmation of a diagnosis of MODYand defines the subgroup. Differences in prognosis and treatment strongly support the increased use of molecular genetic testing in diabetes.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Basic Helix-Loop-Helix Leucine Zipper Transcription Factors
  • Birth Weight
  • DNA-Binding Proteins / genetics
  • Diabetes Mellitus, Type 2 / genetics*
  • Female
  • Glucokinase / genetics
  • Hepatocyte Nuclear Factor 1
  • Hepatocyte Nuclear Factor 1-alpha
  • Hepatocyte Nuclear Factor 1-beta
  • Hepatocyte Nuclear Factor 4
  • Homeodomain Proteins / genetics
  • Humans
  • Islets of Langerhans
  • Male
  • Mutation
  • Nuclear Proteins*
  • Phosphoproteins / genetics
  • Pregnancy
  • Receptors, Cytoplasmic and Nuclear / genetics
  • Trans-Activators / genetics
  • Transcription Factors / genetics

Substances

  • Basic Helix-Loop-Helix Leucine Zipper Transcription Factors
  • DNA-Binding Proteins
  • HNF1A protein, human
  • HNF1B protein, human
  • HNF4A protein, human
  • Hepatocyte Nuclear Factor 1-alpha
  • Hepatocyte Nuclear Factor 4
  • Homeodomain Proteins
  • MLX protein, human
  • Nuclear Proteins
  • Phosphoproteins
  • Receptors, Cytoplasmic and Nuclear
  • Trans-Activators
  • Transcription Factors
  • pancreatic and duodenal homeobox 1 protein
  • Hepatocyte Nuclear Factor 1
  • Hepatocyte Nuclear Factor 1-beta
  • Glucokinase