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

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.