Glycogen storage disease type IV: inherited deficiency of branching enzyme activity in cats

Pediatr Res. 1992 Dec;32(6):719-25. doi: 10.1203/00006450-199212000-00020.

Abstract

Glycogen storage disease type IV due to branching enzyme deficiency was found in an inbred family of Norwegian forest cats, an uncommon breed of domestic cats. Skeletal muscle, heart, and CNS degeneration were clinically apparent and histologically evident in affected cats older than 5 mo of age, but cirrhosis and hepatic failure, hallmarks of the human disorder, were absent. Beginning at or before birth, affected cats accumulated an abnormal glycogen in many tissues that was determined by histochemical, enzymatic, and spectral analysis to be a poorly branched alpha-1,4-D-glucan. Branching enzyme activity was less than 0.1 of normal in liver and muscle of affected cats and partially deficient (0.17-0.75 of normal) in muscle and leukocytes of the parents of affected cats. These data and pedigree analysis indicate that branching enzyme deficiency is a simple autosomal recessive trait in this family. This is the first reported animal model of human glycogen storage disease type IV. A breeding colony derived from a relative of the affected cats has been established.

Publication types

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

MeSH terms

  • 1,4-alpha-Glucan Branching Enzyme / deficiency*
  • Animals
  • Cat Diseases / enzymology
  • Cat Diseases / genetics*
  • Cat Diseases / pathology
  • Cats
  • Female
  • Genes, Recessive
  • Glycogen / metabolism
  • Glycogen Storage Disease Type IV / enzymology
  • Glycogen Storage Disease Type IV / genetics
  • Glycogen Storage Disease Type IV / veterinary*
  • Inclusion Bodies / pathology
  • Male
  • Muscles / pathology
  • Nervous System / pathology
  • Pedigree

Substances

  • Glycogen
  • 1,4-alpha-Glucan Branching Enzyme