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Myelin pp 277-285 | Cite as

Intrathecal Delivery of Viral Vectors for Gene Therapy

Protocol
Part of the Methods in Molecular Biology book series (MIMB, volume 1791)

Abstract

Gene delivery to the peripheral nervous system for therapeutic applications remains technically challenging but could eventually have a significant impact on the development of innovative treatments not only for inherited but also for acquired peripheral neuropathies. Here we describe the method for lumbar intrathecal injection of viral vectors in experimental mice. This gene delivery route provides widespread and stable over time Schwann cell-targeted or ubiquitous gene expression in the peripheral nervous system.

Key words

Gene delivery Gene therapy Lentiviral vectors Intrathecal injection Schwann cells Spinal roots Peripheral nerves 

Notes

Acknowledgments

This work was supported by the Muscular Dystrophy Association (Grants MDA 277250 and 480030 to KAK), by the Charcot-Marie-Tooth Association, and by AFM-Telethon (Grant 19719 to KAK).

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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Neuroscience Laboratory, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular MedicineNicosiaCyprus
  2. 2.Neurology Clinics, The Cyprus Institute of Neurology and Genetics and Cyprus School of Molecular MedicineNicosiaCyprus

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