Cellular pathology and pathogenic aspects of neuronal ceroid lipofuscinoses

Adv Genet. 2001:45:35-68. doi: 10.1016/s0065-2660(01)45003-6.

Abstract

Lysosomal accumulation of autofluorescent, ceroid lipopigment material in various tissues and organs is a common feature of the neuronal ceroid lipofuscinoses (NCLs). However, recent clinicopathologic and genetic studies have evidenced that NCLs encompass a group of highly heterogeneous disorders. In five of the eight NCL variants distinguished at present, genes associated with the disease process have been isolated and characterized (CLN1, CLN2, CLN3, CLN5, CLN8). Only products of two of these genes, CLN 1 and CLN2, have structural and functional properties of lysosomal enzymes. Nevertheless, according to the nature of the material accumulated in the lysosomes, NCLs in humans as well as natural animal models of these disorders can be divided into two major groups: those characterized by the prominent storage of saposins A and D, and those showing the predominance of subunit c of mitochondrial ATP synthase accumulation. Thus, taking into account the chemical character of the major component of the storage material, NCLs can be classified currently as proteinoses. Of importance, although lysosomal storage material accumulates in NCL subjects in various organs, only brain tissue shows severe dysfunction and cell death, another common feature of the NCL disease process. However, the relation between the genetic defects associated with the NCL forms, the accumulation of storage material, and tissue damage is still unknown. This chapter introduces the reader to the complex pathogenesis of NCLs and summarizes our current knowledge of the potential consequences of the genetic defects of NCL-associated proteins on the biology of the cell.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Aminopeptidases
  • Animals
  • Antigens, CD
  • Child
  • Child, Preschool
  • Dipeptidyl-Peptidases and Tripeptidyl-Peptidases
  • Endopeptidases / deficiency
  • Humans
  • Infant
  • Infant, Newborn
  • Lysosomal Membrane Proteins
  • Membrane Glycoproteins / deficiency
  • Neuronal Ceroid-Lipofuscinoses / diagnosis*
  • Neuronal Ceroid-Lipofuscinoses / genetics*
  • Neuronal Ceroid-Lipofuscinoses / pathology*
  • Serine Proteases
  • Thiolester Hydrolases / deficiency
  • Tripeptidyl-Peptidase 1

Substances

  • Antigens, CD
  • Lysosomal Membrane Proteins
  • Membrane Glycoproteins
  • Tripeptidyl-Peptidase 1
  • Thiolester Hydrolases
  • palmitoyl-protein thioesterase
  • Endopeptidases
  • Serine Proteases
  • Aminopeptidases
  • Dipeptidyl-Peptidases and Tripeptidyl-Peptidases
  • TPP1 protein, human