大池
鞘内
中枢神经系统
医学
生物
神经科学
医学物理学
外科
脑脊液
作者
Nathan Katz,Tamara Goode,Christian Hinderer,Juliette Hordeaux,James M. Wilson
出处
期刊:Human Gene Therapy Methods
[Mary Ann Liebert]
日期:2018-07-21
卷期号:29 (5): 212-219
被引量:20
标识
DOI:10.1089/hgtb.2018.041
摘要
Intrathecal delivery of adeno-associated virus vectors and other therapeutics are currently being evaluated for the treatment of central nervous system sequelae of lysosomal storage diseases, motor neuron diseases, and neurodegenerative diseases. As products transition from preclinical to clinical studies, a standardized and clinically relevant method of intrathecal delivery is increasingly germane. Here, we describe a method of intrathecal delivery via suboccipital puncture into the cisterna magna under fluoroscopic guidance in nonhuman primates. This procedure is suitable for use in good laboratory practice compliant studies, has an excellent safety profile, and is highly similar to the procedure currently being explored for use in humans.
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