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Gene therapy administration routes

Systemic administration (intravenous) targets the central and the peripheral nervous system and is the only effective route to target the large and anatomically complex muscle tissue effectively. Intrathecal administration allows targeting of the brain, spinal cord, and peripheral nerves, whereas administration through unique anatomical locations, including directly to the brain parenchyma, facilitates specific targeting of the CNS. (Contributed by Dr. Rotem Orbach. Created with BioRender.com.)

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