Adeno-associated virus gene transfer to mouse retina
Adeno-associated virus gene transfer to mouse retina
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DOI：
10.1089/hum.1998.9.1-81
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发表时间：
1998-01-01
影响因子：
4.2
通讯作者：
Thrasher, AJ
中科院分区：
文献类型：
--
作者：
Ali, RR;Reichel, MB;Thrasher, AJ
眼基因转移可能提供了一种手段，以阻止视网膜变性特征的许多遗传性失明原因，包括视网膜色素变性（RP）。先前，我们已经在免疫缺陷动物中发现，重组腺相关病毒（rAAV）在视网膜下注射后介导光感受器和视网膜色素上皮（RPE）的转导。在这项研究中，我们扩展了这些观察结果，并表明编码报告基因LacZ的高纯化重组AAV载体在视网膜下注射后可以在免疫小鼠品系中转导光感受器，并在玻璃体内注射后有效转导神经节细胞。转导水平随着时间的推移而增加。亚致死的伽马射线照射可促进这一过程。
Ocular gene transfer may provide a means for arresting the retinal degeneration characteristic of many inherited causes of blindness, including retinitis pigmentosa (RP). Previously, we have shown in immunodeficient animals that recombinant adeno-associated virus (rAAV) mediates transduction of photoreceptors as well as the retinal pigment epithelium (RPE) following subretinal injection. In this study we extend these observations and show that highly purified recombinant AAV vectors encoding the reporter gene LacZ transduce photoreceptors in an immunocompetent mouse strain following subretinal injection and efficiently transduce ganglion cells after intravitreal injection. Levels of transduction increase over time. Sublethal gamma-irradiation is shown to facilitate this process.