
Intravenous AAV9 administration results in safe and widespread distribution of transgene in the brain of mini-pig
Brief intro:
- Author: 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
- Journal: Front Cell Dev Biol
- Doi: https://www.doi.org/10.3389/fcell.2022.1115348
- Publication Date: 2023 Jan 24
Products/Services used in the paper
Quotation shows PackGene:GFP-expressing viral vector was obtained from Addgene (plasmid# 67634). The vector was packaged by PackGene Biotech with the AAV9 serotype.
Research Field:gene therapy using mini-pig as model
AAV Serotype:AAV9
Targeted organ:brain
Animal or cell line strain:Bama pigs
Abstract
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.
About PackGene
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
