Stable and efficient gene transfer into the mutant retinal pigment epithelial cells of the Mitfvit mouse using a lentiviral vector
Stable and efficient gene transfer into the mutant retinal pigment epithelial cells of the Mitfvit mouse using a lentiviral vector
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DOI：
10.1076/ceyr.18.2.135.5376
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发表时间：
1999-02-01
影响因子：
2
通讯作者：
Smith, SB
中科院分区：
文献类型：
--
作者：
Galileo, DS;Hunter, K;Smith, SB
目的。本研究的目的是测试在人CMV启动子控制下编码标记LacZ基因的慢病毒载体能否稳定地感染白癜风小鼠的大量RPE细胞。这只小鼠在RPE细胞中含有小眼球基因突变，导致光感受器细胞缓慢进行性退化。将浓缩慢病毒载体HR‘CMVlacZ注射到新生白癜风小鼠的玻璃体内。在注射后2个月的不同时间点处死小鼠，用组织化学方法检测LacZ的表达。慢病毒载体主要感染RPE，并导致LacZ在大量RPE细胞中表达。LacZ在白癜风RPE细胞中的表达至少在两个月内保持稳定。这些结果提出了使用类似的慢病毒载体将小眼炎基因的正确拷贝导入RPE的可能性，最终可能挽救这一突变小鼠的光感受器细胞。
Purpose. The purpose of the present study was to test whether a lentiviral vector encoding the marker lacZ gene under the control of the human CMV promoter would stably infect a significant number of RPE cells in the vitiligo mouse. This mouse harbors a mutation in the microphthalmia gene in RPE cells that leads to slow progressive photoreceptor cell degeneration.Methods. Concentrated lentiviral vector HR'CMVlacZ was injected intravitreally into newborn vitiligo mice. Mice were sacrificed at various time points up to two months post-injection and eyes were processed histochemically to detect lacZ expression.Results. The lentiviral vector infected predominantly the RPE and resulted in lacZ expression in numerous RPE cells at all times analyzed.Conclusions. LacZ expression in vitiligo RPE cells appeared to be stable for a period of at least two months. These results raise the possibility of using a similar lentiviral vector for introduction of a correct copy of the microphthalmia cDNA into the RPE that may ultimately rescue photoreceptor cells in this mutant mouse.