AAV AS VECTOR FOR TREATMENT OF GLOBOID CELL LEUKODYSTROPHY

AAV载体作为基因治疗载体显示出巨大的应用前景。我们 正在开发对治疗恒河猴有用的载体 患有球状细胞白质营养不良。我们给两只幼年恒河猴接种了疫苗 携带绿色荧光蛋白AAV载体的猴子 报告基因在肌肉内和大脑内。两人的活组织检查 动物在肌肉中表现出少量的荧光。一只猴子 因收集组织而被安乐死。少量的 在脑内针道附近可观察到荧光。 到目前为止，这项初步研究已经证明了使用 这些猴子体内的AAV媒介。我们计划把剩下的地方重新安置起来 猴子评估在原始接种地点的持久性和 第二次接种病媒的效果。资助基础赠款， 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