Intrathecal Delivery of AAV Vectors in Cynomolgus Macaques for CNS Gene Therapy and Gene Expression Analysis in Microdissected Motor Neurons

  • Florie BorelEmail author
  • Eric Adams
  • Christian MuellerEmail author
Part of the Methods in Molecular Biology book series (MIMB, volume 1937)


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.

Key words

AAV Adeno-associated virus Intrathecal Trendelenburg CSF CNS Laser-capture microdissection Motor neurons ddPCR 



Florie Borel would like to express her gratitude to Rachel Stock from the Mueller Lab at UMass Medical for generating the images of the laser capture, as well as to Christen Simon and Randy Reed at Northern Biomedical Research for their support.


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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2019

Authors and Affiliations

  1. 1.Gene Therapy CenterUniversity of Massachusetts Medical SchoolWorcesterUSA
  2. 2.Northern Biomedical ResearchNorton ShoresUSA
  3. 3.Department of PediatricsUniversity of Massachusetts Medical SchoolWorcesterUSA

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