GENE-THERAPY VIA PRIMARY MYOBLASTS - LONG-TERM EXPRESSION OF FACTOR-IX PROTEIN FOLLOWING TRANSPLANTATION INVIVO

被引:269
|
作者
DAI, Y
ROMAN, M
NAVIAUX, RK
VERMA, IM
机构
[1] SALK INST,MOLEC BIOL & VIROL LAB,POB 85800,SAN DIEGO,CA 92186
[2] UNIV CALIF SAN DIEGO,SCH MED,DEPT PEDIAT,LA JOLLA,CA 92093
[3] UNIV CALIF SAN DIEGO,SCH MED,CTR MOLEC GENET,LA JOLLA,CA 92093
关键词
RETROVIRAL VECTORS; TISSUE-SPECIFIC ENHANCER; MUSCLE;
D O I
10.1073/pnas.89.22.10892
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
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.
引用
收藏
页码:10892 / 10895
页数:4
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