Kaleko M, Garcia J V, Miller A D
Program in Molecular Medicine, Fred Hutchinson Cancer Research Center, Seattle, WA 98104.
Hum Gene Ther. 1991 Spring;2(1):27-32. doi: 10.1089/hum.1991.2.1-27.
The development of liver-directed gene therapy protocols depends upon the ability to transfer genes into a large number of liver cells such that the genes are expressed persistently. We used a retroviral vector to transfer the gene for neomycin phosphotransferase (neo) into mouse liver cells in vivo. Direct injection of the retrovirus preparation into mitotically active (regenerating) liver parenchyma resulted in efficient gene transfer, with neo sequences detectable in the livers of every animal tested 10 weeks to 6 months later. The neo gene was expressed for at least 3 months. This methodology may eventually be applicable to the treatment of human disease.
肝脏定向基因治疗方案的发展取决于将基因导入大量肝细胞并使其持续表达的能力。我们使用逆转录病毒载体在体内将新霉素磷酸转移酶（neo）基因导入小鼠肝细胞。将逆转录病毒制剂直接注射到有丝分裂活跃（再生）的肝实质中可实现高效的基因转移，在10周龄至6月龄的每只受试动物肝脏中均能检测到neo序列。neo基因至少表达了3个月。这种方法最终可能适用于人类疾病的治疗。
