Abstract
Gene therapy shows great promise for the treatment of neurological disorders, and accessing cerebrospinal fluid (CSF) from the cerebellomedullary cistern through the posterior atlanto-occipital membrane has become a common route of delivery in preclinical studies. Unlike direct brain parenchymal infusions, CSF delivery offers broader coverage to the central and peripheral nervous system. This prospectively increases its translational value, more specially to treat global brain dysfunctions in which the pathology is disseminated throughout the brain and not focalized in one specific brain structure. Also, from the practical point of view, this approach offers a more reliable method for neurological gene replacement in infants, whose immature cranial suture preclude the use of skull-mounted devices. Here we describe a consistent, precise, and safe method for CSF injection.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
References
Foust KD, Nurre E, Montgomery CL et al (2009) Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 27:59–65. https://doi.org/10.1038/nbt.1515
Samaranch L, Salegio EA, San Sebastian W et al (2013) Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates. Hum Gene Ther 24:526–532. https://doi.org/10.1089/hum.2013.005
Samaranch L, Salegio EA, San Sebastian W et al (2012) Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum Gene Ther 23:382–389. https://doi.org/10.1089/hum.2011.200
Salegio EA, Streeter H, Dube N et al (2014) Distribution of nanoparticles throughout the cerebral cortex of rodents and non-human primates: implications for gene and drug therapy. Front Neuroanat 8:9. https://doi.org/10.3389/fnana.2014.00009
Acknowledgments
This work was supported by a grant to K.S.B. from NIH-NINDS (R01NS073940-01).
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2019 Springer Science+Business Media, LLC, part of Springer Nature
About this protocol
Cite this protocol
Samaranch, L., Ohno, K., San Sebastian, W., Bankiewicz, K. (2019). Cerebellomedullary Cistern Injection of Viral Vectors in Nonhuman Primates. In: Manfredsson, F., Benskey, M. (eds) Viral Vectors for Gene Therapy. Methods in Molecular Biology, vol 1937. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-9065-8_21
Download citation
DOI: https://doi.org/10.1007/978-1-4939-9065-8_21
Published:
Publisher Name: Humana Press, New York, NY
Print ISBN: 978-1-4939-9064-1
Online ISBN: 978-1-4939-9065-8
eBook Packages: Springer Protocols