鱼眼病患者的载脂蛋白 A-I 相关脂蛋白代谢紊乱，通过体外基因转染的脂肪细胞产生的卵磷脂：胆固醇酰基转移酶可得到改善。
Disturbed apolipoprotein A-I-containing lipoproteins in fish-eye disease are improved by the lecithin:cholesterol acyltransferase produced by gene-transduced adipocytes in vitro.
机构信息
Department of Genome Research and Clinical Application, Graduate School of Medicine, Chiba University, 1-8-1 Inohana, Chiba, Japan.
出版信息
Mol Genet Metab. 2011 Feb;102(2):229-31. doi: 10.1016/j.ymgme.2010.10.009. Epub 2010 Oct 20.
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.
我们报告了通过 lcat 基因转导增殖脂肪细胞（ccdPA/lcat）产生的重组 LCAT 的体外功效，该 LCAT 已被开发用于酶替代疗法。用 apoA-I 特异性免疫检测结合 1D 和 2D 凝胶电泳显示，用 ccdPA/lcat 衍生的重组 LCAT 体外孵育可明显改善高密度脂蛋白亚群谱的紊乱。因此，这些使用 ccdPA/lcat 的结果强烈表明细胞植入可以为 LCAT 缺乏症患者提供酶替代治疗。
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