Strauss M
Max-Planck-Gesellschaft, Humboldt Universität, Max-Delbrück-Center for Molecular Medicine, Berlin-Buch, Germany.
Gene Ther. 1994 May;1(3):156-64.
The development of strategies for gene therapy in the liver is a challenging task because this organ is particularly involved in the manifestation of numerous genetic diseases as well as in malignant and infectious diseases. Most of the available techniques for gene transfer were already applied to hepatocytes ex vivo. Attempts to retransplant retrovirally transduced hepatocytes back to patients have shown the limits of the ex vivo approach. The obvious alternative would be the development of suitable techniques for gene transfer in vivo. Both viral and non-viral delivery systems were tested in animal models with limited success. This article summarizes the state of the art of the available techniques and vectors and discusses the essential future developments.
肝脏基因治疗策略的开发是一项具有挑战性的任务，因为该器官特别涉及多种遗传疾病以及恶性和感染性疾病的表现。大多数现有的基因转移技术已应用于离体肝细胞。将逆转录病毒转导的肝细胞重新移植回患者体内的尝试已经显示出离体方法的局限性。显而易见的替代方案是开发适用于体内基因转移的技术。病毒和非病毒递送系统都在动物模型中进行了测试，但取得的成功有限。本文总结了现有技术和载体的最新状况，并讨论了未来的关键发展方向。
