AAV AS VECTOR FOR TREATMENT OF GLOBOID CELL LEUKODYSTROPHY

AAV 作为治疗球状细胞脑白质营养不良的载体

• 批准号: 6116208
• 负责人: GARY B BASKIN
• 金额: $ 16.14万
• 依托单位: TULANE UNIVERSITY OF LOUISIANA
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-05-01 至 2000-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6116208
• 关键词: Mammalia; biotechnology; congenital disorders; gene therapy; inborn metabolism disorder; model design /development; technology /technique; virus

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

AAV矢量作为基因疗法的向量显示出巨大的希望。 我们 正在开发矢量，这对于治疗恒河猴很有用 与球形细胞白细胞营养不良。 我们接种了2个婴儿恒河 猴子具有含有绿色荧光蛋白的AAV载体的猴子 报告基因肌肉内和脑内。 两者的活检 动物在肌肉中显示出少量的荧光。 一只猴子 被安乐死以进行组织收集。 少量 观察到荧光与大脑的针刺相邻。 迄今为止，这项试验研究已经证明了使用 这些猴子中的AAV矢量。 我们计划重新启动剩余的 猴子评估原始接种站点的持久性和 第二个载体给药的影响。 资金基础赠款， 风险研究出版物无