在LDLR缺乏的子中,ex vivo基因治疗后高胆固醇血症的长期改善
J R Chowdhury1, M Grossman, S Gupta
1Department of Internal Medicine, Albert Einstein College of Medicine, Bronx, NY 10461.
Science (New York, N.Y.)
|December 30, 1991
概括
家庭高胆固醇血症 (FH) 基因疗法使用纠正的肝细胞降低了子的胆固醇. 这种方法对治疗遗传性高胆固醇疾病充满希望.
科学领域:
- 生物医学研究的研究.
- 基因治疗是一种基因疗法.
- 肝病学 肝病学是一种肝病学.
背景情况:
- 家族性高胆固醇血症 (FH) 是一种遗传性疾病,由于低密度脂蛋白受体 (LDLR) 缺乏,导致胆固醇高.
- 瓦塔纳贝遗传性高脂血症子作为FH研究的动物模型.
研究的目的:
- 开发和评估用于FH的肝脏基因疗法.
- 评估自主肝细胞的ex vivo基因校正疗效.
主要方法:
- 利用复合逆转录病毒进行子肝细胞的ex vivo遗传校正.
- 在FH子中移植了转基因原生肝细胞.
- 在移植后监测血清胆固醇水平和LDLRRNA表达.
主要成果:
- 移植后的子在122天内显示出血清总胆固醇持续下降30-50%.
- 再组合衍生的LDLRRNA在移植后长达6.5个月的组织中可检测,没有显著下降.
结论:
- 自体肝细胞的ex vivo基因校正是FH肝脏基因治疗的可行策略.
- 这种方法有效地降低了FH动物模型中的血清胆固醇.
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