Disturbed apolipoprotein A-I-containing lipoproteins in fish-eye disease are improved by the lecithin:cholesterol acyltransferase produced by gene-transduced adipocytes in vitro

Mol Genet Metab. 2011 Feb;102(2):229-31. doi: 10.1016/j.ymgme.2010.10.009. Epub 2010 Oct 20.

Abstract

We report the in vitro efficacy of recombinant LCAT produced by lcat gene-transduced proliferative adipocytes (ccdPA/lcat), which has been developed for enzyme replacement therapy. ApoA-I-specific immunodetection in combination with 1D and 2D gel electrophoreses showed that the disturbed high-density lipoprotein subpopulation profile was clearly ameliorated by the in vitro incubation with ccdPA/lcat-derived recombinant LCAT. Thus, these results using ccdPA/lcat strongly suggest the cell implantation could contribute the enzyme replacement for the patients with LCAT deficiency.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adipocytes / enzymology*
  • Apolipoprotein A-I / genetics*
  • Apolipoprotein A-I / metabolism*
  • Cells, Cultured
  • Gene Transfer Techniques
  • Genetic Vectors / genetics
  • Humans
  • In Vitro Techniques
  • Lecithin Cholesterol Acyltransferase Deficiency / enzymology*
  • Lecithin Cholesterol Acyltransferase Deficiency / genetics*
  • Male
  • Phosphatidylcholine-Sterol O-Acyltransferase / blood
  • Phosphatidylcholine-Sterol O-Acyltransferase / genetics
  • Phosphatidylcholine-Sterol O-Acyltransferase / metabolism*

Substances

  • Apolipoprotein A-I
  • Phosphatidylcholine-Sterol O-Acyltransferase