Standardized Method for Intra-Cisterna Magna Delivery Under Fluoroscopic Guidance in Nonhuman Primates.

Q1 Immunology and Microbiology Human Gene Therapy Methods Pub Date : 2018-10-01 Epub Date: 2018-08-14 DOI:10.1089/hgtb.2018.041
Nathan Katz, Tamara Goode, Christian Hinderer, Juliette Hordeaux, James M Wilson
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引用次数: 16

Abstract

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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非人类灵长类动物在透视引导下大池内输送的标准化方法。
鞘内递送腺相关病毒载体和其他治疗方法目前正在评估用于治疗溶酶体贮积病、运动神经元疾病和神经退行性疾病的中枢神经系统后遗症。随着产品从临床前研究向临床研究过渡,一种标准化且与临床相关的鞘内给药方法日益迫切。在这里,我们描述了一种在非人类灵长类动物中在透视引导下通过枕下穿刺进入大池的鞘内分娩方法。该程序适用于符合良好实验室规范的研究,具有极好的安全性,并且与目前正在探索的用于人体的程序高度相似。
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来源期刊
Human Gene Therapy Methods
Human Gene Therapy Methods BIOTECHNOLOGY & APPLIED MICROBIOLOGY-GENETICS & HEREDITY
CiteScore
5.80
自引率
0.00%
发文量
0
审稿时长
>12 weeks
期刊介绍: Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases. The Journal is divided into three parts. Human Gene Therapy, the flagship, is published 12 times per year. HGT Methods, a bimonthly journal, focuses on the applications of gene therapy to product testing and development. HGT Clinical Development, a quarterly journal, serves as a venue for publishing data relevant to the regulatory review and commercial development of cell and gene therapy products.
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