Polycystic disease of the liver

Hepatology. 2004 Oct;40(4):774-82. doi: 10.1002/hep.20431.

Abstract

Autosomal dominant polycystic disease is genetically heterogeneous with mutations in two distinct genes predisposing to the combination of renal and liver cysts (AD-PKD1 and AD-PKD2) and mutations in a third gene yielding isolated liver cysts (the polycystic liver disease gene). Transcription and translation of the PKD1 gene produces polycystin-1, an integral membrane protein that may serve as an extracellular receptor. Mutations occur throughout the PKD1 gene, but more severe disease is associated with N-terminal mutations. The PKD2 gene product, polycystin-2, is an integral membrane protein with molecular characteristics of a calcium-permeant cation channel. Mutations occur throughout the PKD2 gene, and severity of disease may vary with site of mutation in PKD2 and the functional consequence on the resultant polycystin-2 protein. Polycystic liver disease is genetically linked to protein kinase C substrate 80K-H (PRKCSH). The PRKCSH gene encodes hepatocystin, a protein that moderates glycosylation and fibroblast growth factor receptor signaling. More prominent in women, hepatic cysts emerge after the onset of puberty and dramatically increase in number and size through the child-bearing years of early and middle adult life. Although liver failure or complications of advanced liver disease are rare, some patients develop massive hepatic cystic disease and become clinically symptomatic. There is no effective medical therapy. Interventional and surgical options include cyst aspiration and sclerosis, open or laparoscopic cyst fenestration, hepatic resection, and liver transplantation.

Publication types

  • Review

MeSH terms

  • Humans
  • Membrane Proteins / genetics
  • Membrane Proteins / physiology*
  • Polycystic Kidney Diseases* / diagnosis
  • Polycystic Kidney Diseases* / genetics
  • Polycystic Kidney Diseases* / therapy
  • Proteins / genetics
  • Proteins / physiology*
  • TRPP Cation Channels

Substances

  • Membrane Proteins
  • Proteins
  • TRPP Cation Channels
  • polycystic kidney disease 1 protein
  • polycystic kidney disease 2 protein