Prolonged expression of therapeutic levels of human granulocyte colony-stimulating factor in rats following gene transfer to skeletal muscle

被引:17
作者
Bonham, L [1 ]
Palmer, T [1 ]
Miller, AD [1 ]
机构
[1] FRED HUTCHINSON CANC RES CTR,SEATTLE,WA 98109
关键词
D O I
10.1089/hum.1996.7.12-1423
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
Gene transfer to skeletal muscle was examined as a means of gene therapy for neutropenia, A recombinant retrovirus containing a human granulocyte colony-stimulating factor (G-CSF) gene was introduced into primary human or rat myoblasts, which were then shown to produce biologically active G-CSF. Transplantation of G-CSF-producing rat myoblasts into the muscle of syngeneic rats resulted in a 15-fold increase in absolute neutrophil counts, This increase correlated with detection of circulating human G-CSF protein throughout the 6-month duration of the experiment. These results clearly demonstrate long-term production of therapeutically relevant amounts of a human protein by normal cells in vivo.
引用
收藏
页码:1423 / 1429
页数:7
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