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.
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Acknowledgments
This work was supported by a grant to K.S.B. from NIH-NINDS (R01NS073940-01).
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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
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DOI: https://doi.org/10.1007/978-1-4939-9065-8_21
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Publisher Name: Humana Press, New York, NY
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Online ISBN: 978-1-4939-9065-8
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