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载体作为基因治疗载体具有广阔的应用前景。我们