Front Cell Dev Biol. 2023 Jan 24
Yingqi Lin, Caijuan Li, Wei Wang, Jiawei Li, Chunhui Huang, Xiao Zheng, Zhaoming Liu, Xichen Song, Yizhi Chen, Jiale Gao, Jianhao Wu, Jiaxi Wu, Zhuchi Tu, Liangxue Lai, Xiao-Jiang Li, Shihua Li, and Sen Yan
Products used in the paper Details Operation
AAV vector packaging GFP-expressing viral vector was obtained from Addgene (plasmid# 67634). The vector was packaged by PackGene Biotech with the AAV9 serotype. Request Quote

Research Field: gene therapy using mini-pig as model

Keywords: AAV9, CNS, large animal, pig, neurodegeneration

AAV Serotype: AAV9

Dose: Viruses (300 ml of 10^13 vg/ml diluted in 1 ml saline) or the same volume of saline were injected through the auricular vein into each pig over the course of 5 min.

Routes of Administration: auricular vein

Targeted organ: brain

Animal or cell line strain: Bama pigs


Animal models are important for understanding the pathogenesis of human diseases and for developing and testing new drugs. Pigs have been widely used in the research on the cardiovascular, skin barrier, gastrointestinal, and central nervous systems as well as organ transplantation. Recently, pigs also become an attractive large animal model for the study of neurodegenerative diseases because their brains are very similar to human brains in terms of mass, gully pattern, vascularization, and the proportions of the gray and white matters. Although adeno-associated virus type 9 (AAV9) has been widely used to deliver transgenes in the brain, its utilization in large animal models remains to be fully characterized. Here, we report that intravenous injection of AAV9-GFP can lead to widespread expression of transgene in various organs in the pig. Importantly, GFP was highly expressed in various brain regions, especially the striatum, cortex, cerebellum, hippocampus, without detectable inflammatory responses. These results suggest that intravenous AAV9 administration can be used to establish large animal models of neurodegenerative diseases caused by gene mutations and to treat these animal models as well.

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