Zhou S, Murphy J E, Escobedo J A, Dwarki V J
Chiron Corporation, Emeryville, CA, USA.
Gene Ther. 1998 May;5(5):665-70. doi: 10.1038/sj.gt.3300648.
Recombinant adeno-associated virus encoding the monkey erythropoietin gene (rAAV-cm-Epo) was generated and tested for its potential to confer long-term expression of the gene product following intramuscular injection. A single intramuscular injection of 2 x 10(12) rAAV-cm-Epo particles into two baboons led to sustained high circulating Epo levels and a concomitant increase in hematocrit. The hematocrits reached 62 and 75% by week 10 (from pre-injection values of 38 and 40%, respectively) and remained elevated throughout the study period (28 weeks). Circulating Epo levels were also elevated throughout the study period. Our data demonstrate the potential for long-term gene expression in large animals by a single intramuscular injection of a recombinant adeno-associated virus (rAAV) vector.
构建了编码猴促红细胞生成素基因的重组腺相关病毒（rAAV-cm-Epo），并对其在肌肉注射后赋予基因产物长期表达的潜力进行了测试。向两只狒狒单次肌肉注射2×10¹²个rAAV-cm-Epo颗粒，导致促红细胞生成素（Epo）在循环中的水平持续升高，同时血细胞比容也随之增加。到第10周时，血细胞比容分别从注射前的38%和40%升至62%和75%，并在整个研究期间（28周）一直保持升高。在整个研究期间，循环中的Epo水平也一直升高。我们的数据证明了通过单次肌肉注射重组腺相关病毒（rAAV）载体在大型动物中实现长期基因表达的潜力。
