Raper S E
Department of Surgery, University of Pennsylvania School of Medicine, Philadelphia, USA.
Clin Transplant. 1995 Jun;9(3 Pt 2):249-54.
Trials of liver-directed gene therapy are a clinical reality. Technology is rapidly developing for the isolation of large numbers of human hepatocytes. Recombinant DNA techniques are available to engineer recombinant retroviruses which express genes of clinical relevance. Familial hypercholesterolemia serves as the clinical paradigm for liver-directed gene therapy, and the clinical protocol is underway. Early results are promising. Techniques for hepatocyte delivery, optimization of gene transfer, and safety considerations are important considerations in designing a human trial. Other diseases which are likely to be among the first treated with this new approach are ornithine transcarbamylase deficiency, hemophilia B, and Crigler-Najjar syndrome.
肝脏定向基因治疗试验已成为临床现实。用于分离大量人类肝细胞的技术正在迅速发展。重组DNA技术可用于构建表达具有临床相关性基因的重组逆转录病毒。家族性高胆固醇血症是肝脏定向基因治疗的临床范例，临床方案正在进行中。早期结果很有前景。在设计人体试验时，肝细胞递送技术、基因转移优化和安全考虑是重要的考量因素。可能首先采用这种新方法治疗的其他疾病包括鸟氨酸转氨甲酰酶缺乏症、乙型血友病和克里格勒-纳贾尔综合征。
