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通过脑室内给药,在小鼠脑室区域实现超声响应纳米泡介导的基因转染。

Ultrasound-responsive nanobubble-mediated gene transfection in the cerebroventricular region by intracerebroventricular administration in mice.

机构信息

Graduate School of Biomedical Sciences, Nagasaki University, 1-7-1 Sakamoto, Nagasaki-shi, Nagasaki 852-8588, Japan.

Graduate School of Biomedical Sciences, Nagasaki University, 1-7-1 Sakamoto, Nagasaki-shi, Nagasaki 852-8588, Japan.

出版信息

Eur J Pharm Biopharm. 2019 Apr;137:1-8. doi: 10.1016/j.ejpb.2019.02.003. Epub 2019 Feb 7.

DOI:10.1016/j.ejpb.2019.02.003
PMID:30738859
Abstract

AIM

Intracerebroventricular (ICV) administration of ultrasound-responsive bubbles and cranial ultrasound irradiation is reported as a transfection system for the cerebroventricular region. This study aimed to characterize the transfection system with respect to transfection efficiency, spatial distribution of transgene expression, and safety.

METHODS

Plasmid DNA was transfected to mouse brain by ICV injection of ultrasound-responsive nanobubbles, followed by ultrasound irradiation to brain. Spatial distribution of transgene expression in the cerebroventricular region was investigated using multicolor deep imaging.

RESULT

This transfection system efficiently transferred the transgene to the choroid plexus with no morphological change or cerebral hemorrhage. Moreover, sustained secretion of transgenic protein was achieved by transferring the transgene encoding the secretable protein.

CONCLUSION

We successfully developed an ultrasound-responsive nanobubbles-mediated method for gene transfection into the cerebroventricular region via ICV administration in mice.

摘要

目的

脑室内(ICV)给予超声响应性气泡和颅超声辐射被报道为脑室内区域的转染系统。本研究旨在研究转染系统的转染效率、转基因表达的空间分布和安全性。

方法

通过 ICV 注射超声响应性纳米气泡将质粒 DNA 转染到小鼠脑内,然后对大脑进行超声照射。利用多色深度成像研究了转基因在脑室区域的空间分布。

结果

该转染系统可将转基因有效转染到脉络丛,且无形态改变或脑内出血。此外,通过转染编码可分泌蛋白的转基因,实现了转基因蛋白的持续分泌。

结论

我们成功开发了一种通过 ICV 给药在小鼠中进行脑室内基因转染的超声响应性纳米气泡介导方法。

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