作者
Charles H Vite, Marco A Passini, Mark E Haskins, John H Wolfe
发表日期
2003/10
期刊
Gene therapy
卷号
10
期号
22
页码范围
1874-1881
出版商
Nature Publishing Group
简介
Adeno-associated virus (AAV) vectors are capable of delivering a therapeutic gene to the mouse brain that can result in long-term and widespread protein production. However, the human infant brain is more than 1000 times larger than the mouse brain, which will make the treatment of global neurometabolic disorders in children more difficult. In this study, we evaluated the ability of three AAV serotypes (1, 2, and 5) to transduce cells in the cat brain as a model of a large mammalian brain. The human lysosomal enzyme β-glucuronidase (GUSB) was used as a reporter gene, because it can be distinguished from feline GUSB by heat stability. The vectors were injected into the cerebral cortex, caudate nucleus, thalamus, corona radiata, internal capsule, and centrum semiovale of 8-week-old cats. The brains were evaluated for gene expression using in situ hybridization and enzyme histochemistry 10 weeks after …
引用总数
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