AAV AS VECTOR FOR TREATMENT OF GLOBOID CELL LEUKODYSTROPHY

AAV载体作为基因治疗的载体显示出巨大的前景。 我们 正在开发一种载体， 球样细胞脑白质营养不良 我们接种了2只恒河猴幼崽 用含有绿色荧光蛋白的AAV载体 肌内和脑内报告基因。 两人的活检 动物在肌肉中显示少量荧光。 一只猴子 被安乐死以收集组织。 少量的 在脑内邻近针道处观察到荧光。 到目前为止，这项试点研究已经证明了使用 在这些猴子中的AAV载体。 我们计划把剩下的 猴评价初始接种部位的持久性， 第二次施用载体的效果。 基金基础补助金， 风险研究出版物无

AAV vectors show great promise as vectors for gene therapy. We are developing vectors which will be useful to treat rhesus monkeys with globoid cell leukodystrophy. We inoculated 2 infant rhesus monkeys with an AAV vector containing the green fluorescent protein reporter gene intramuscularly and intracerebrally. Biopsies from both animals showed a small amount of fluorescence in muscle. One monkey was euthanatized for tissue collection. A small amount of fluorescence was observed adjacent to the needle tract in the brain. To date, this pilot study has demonstrated the feasability of using an AAV vector in these monkeys. We plan to reinoculate the remaining monkey to evaluate persistence at the original inoculation sites and the effect of a second administration of vector. FUNDING Base Grant, Venture Research PUBLICATIONS None