Intrathecal Delivery of Viral Vectors for Gene Therapy
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 wordsGene delivery Gene therapy Lentiviral vectors Intrathecal injection Schwann cells Spinal roots Peripheral nerves
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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