GENE-THERAPY FOR DIABETES-MELLITUS IN RATS BY HEPATIC EXPRESSION OF INSULIN

被引:120
|
作者
KOLODKA, TM
FINEGOLD, M
MOSS, L
WOO, SLC
机构
[1] BAYLOR COLL MED, DEPT CELL BIOL, HOUSTON, TX 77030 USA
[2] BAYLOR COLL MED, DEPT MED, HOUSTON, TX 77030 USA
[3] BAYLOR COLL MED, DEPT PATHOL, HOUSTON, TX 77030 USA
关键词
IN VIVO GENE THERAPY; RECOMBINANT RETROVIRAL VECTORS; INSULIN GENE EXPRESSION; HEPATIC GENE TRANSFER;
D O I
10.1073/pnas.92.8.3293
中图分类号
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号
07 ; 0710 ; 09 ;
摘要
Type 1 diabetes mellitus is caused by severe insulin deficiency secondary to the autoimmune destruction of pancreatic beta cells. Patients need to be controlled by periodic insulin injections to prevent the development of ketoacidosis, which can be fatal. Sustained, low-level expression of the rat insulin 1 gene from the liver of severely diabetic rats was achieved by in vivo administration of a recombinant retroviral vector. Ketoacidosis was prevented and the treated animals exhibited normoglycemia during a 24-hr fast, with no evidence of hypoglycemia. Histopathological examination of the liver in the treated animals showed no apparent abnormalities. Thus, the liver is an excellent target organ for ectopic expression of the insulin gene as a potential treatment modality for type 1 diabetes mellitus by gene therapy.
引用
收藏
页码:3293 / 3297
页数:5
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