SYSTEMIC DELIVERY OF RECOMBINANT PROTEINS BY GENETICALLY MODIFIED MYOBLASTS
SYSTEMIC DELIVERY OF RECOMBINANT PROTEINS BY GENETICALLY MODIFIED MYOBLASTS
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DOI：
10.1126/science.1962212
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发表时间：
1991-12-06
期刊：
影响因子：
56.9
通讯作者：
LEIDEN, JM
中科院分区：
文献类型：
--
作者：
BARR, E;LEIDEN, JM
将重组蛋白稳定递送至体循环的能力将促进多种获得性和遗传性疾病的治疗。为了探索使用基因工程成肌细胞作为重组蛋白递送系统的可行性，产生了小鼠C2 C12成肌细胞系的稳定转染子，其在体外合成并分泌高水平的人生长激素（hGH）。注射hGH转染的成肌细胞的小鼠在肌肉和血清中均具有显著水平的hGH，其在注射后至少3周保持稳定。用表达β-半乳糖苷酶的C2 C12成肌细胞注射的肌肉的组织学检查表明，许多注射的细胞融合形成多核肌管。因此，基因工程改造的成肌细胞可用于将重组蛋白稳定递送到循环中。
The ability to stably deliver recombinant proteins to the systemic circulation would facilitate the treatment of a variety of acquired and inherited diseases. To explore the feasibility of the use of genetically engineered myoblasts as a recombinant protein delivery system, stable transfectants of the murine C2C12 myoblast cell line were produced that synthesize and secrete high levels of human growth hormone (hGH) in vitro. Mice injected with hGH-transfected myoblasts had significant levels of hGH in both muscle and serum that were stable for at least 3 weeks after injection. Histological examination of muscles injected with beta-galactosidase-expressing C2C12 myoblasts demonstrated that many of the injected cells had fused to form multinucleated myotubes. Thus, genetically engineered myoblasts can bc used for the stable delivery of recombinant proteins into the circulation.