Borel Florie, Adams Eric, Mueller Christian
Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.
Northern Biomedical Research, Norton Shores, MI, USA.
Methods Mol Biol. 2019;1937:295-303. doi: 10.1007/978-1-4939-9065-8_19.
This protocol describes a method of delivering adeno-associated viral (AAV) vectors to the intrathecal space of nonhuman primates for CNS-directed gene therapy. It includes the surgical implantation of the catheter, vector infusion, necropsy, laser-capture microdissection of motor neurons, and gene expression analysis. This method allows efficient and reproducible delivery, and would be of interest to test gene therapy vectors for the treatment of disorders of the central nervous system of nonhuman primates. This protocol was tested in cynomolgus macaques and may be adapted for AAV delivery to different species of large animals.
本方案描述了一种将腺相关病毒（AAV）载体递送至非人灵长类动物鞘内空间以进行中枢神经系统定向基因治疗的方法。它包括导管的手术植入、载体输注、尸检、运动神经元的激光捕获显微切割以及基因表达分析。该方法允许高效且可重复的递送，对于测试用于治疗非人灵长类动物中枢神经系统疾病的基因治疗载体具有重要意义。本方案已在食蟹猕猴中进行了测试，并且可能适用于将AAV递送至不同种类的大型动物。
