The molecular mechanisms of congenital hypofibrinogenaemia

Cell Mol Life Sci. 2004 Jun;61(12):1427-38. doi: 10.1007/s00018-004-3458-8.

Abstract

Congenital hypofibrinogenaemia is characterized by abnormally low levels of fibrinogen and is usually caused by heterozygous mutations in the fibrinogen chain genes (alpha, beta and gamma). However, it does not usually result in a clinically significant condition unless inherited in a homozygous or compound heterozygous state, where it results in a severe bleeding disorder, afibrinogenaemia. Various protein and expression studies have improved our understanding of how mutations causing hypo- and afibrinogenaemia affect secretion of the mature fibrinogen molecule from the hepatocyte. Some mutations can perturb chain assembly as in the gamma153 Cys-->Arg case, while others such as the Bbeta Leu-->Arg and the Bbeta414 Gly-->Ser mutations allow intracellular hexamer assembly but inhibit protein secretion. An interesting group of mutations, such as gamma284 Gly-->Arg and gamma375 Arg-->Trp, not only cause hypofibrinogenaemia but are also associated with liver disease. The nonexpression of these variant chains in plasma fibrinogen is due to retention in the endoplasmic reticulum, which in turn leads to hypofibrinogenaemia.

Publication types

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

MeSH terms

  • Afibrinogenemia / congenital*
  • Afibrinogenemia / genetics*
  • Amino Acid Sequence
  • Endoplasmic Reticulum / metabolism
  • Fibrinogen / biosynthesis
  • Fibrinogen / chemistry
  • Fibrinogen / genetics*
  • Fibrinogen / metabolism
  • Heterozygote
  • Humans
  • Molecular Sequence Data
  • Mutation
  • Protein Conformation
  • Protein Structure, Secondary
  • Protein Structure, Tertiary

Substances

  • Fibrinogen