Dai Y, Roman M, Naviaux R K, Verma I M
Salk Institute, Molecular Biology and Virology Laboratory, San Diego, CA 92186-5800.
Proc Natl Acad Sci U S A. 1992 Nov 15;89(22):10892-5. doi: 10.1073/pnas.89.22.10892.
We have explored the use of primary myoblasts as a somatic tissue for gene therapy of acquired and inherited diseases where systemic delivery of a gene product may have therapeutic effects. Mouse primary myoblasts were infected with replication-defective retroviruses expressing canine factor IX cDNA under the control of a mouse muscle creatine kinase enhancer and human cytomegalovirus promoter. The infected myoblasts were injected into the hindlegs of recipient mice and levels of secreted factor IX protein were monitored in the plasma. We report sustained expression of factor IX protein for over 6 months without any apparent adverse effect on the recipient mice.
我们已经探索了将原代成肌细胞作为一种体细胞组织用于后天性和遗传性疾病的基因治疗，在这些疾病中，基因产物的全身递送可能具有治疗效果。将小鼠原代成肌细胞用复制缺陷型逆转录病毒感染，该病毒在小鼠肌肉肌酸激酶增强子和人巨细胞病毒启动子的控制下表达犬因子IX cDNA。将感染的成肌细胞注射到受体小鼠的后肢，并监测血浆中分泌的因子IX蛋白水平。我们报告了因子IX蛋白持续表达超过6个月，且对受体小鼠没有任何明显的不良影响。
