期刊
MOLECULAR GENETICS AND METABOLISM
卷 102, 期 2, 页码 229-231出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/j.ymgme.2010.10.009
关键词
Lecithin:cholesterol acyltransferase; Fish-eye disease; Familial LCAT deficiency; Apolipoprotein A-I; Protein replacement therapy
资金
- Health and Labour Sciences Research Grants for Translational Research, Japan
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. (C) 2010 Elsevier Inc. All rights reserved.
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