Gene therapy strategies for the treatment of spinal cord injury

Kenzo Uchida*, Hideaki Nakajima, Alexander Rodriguez Guerrero, William Eb Johnson, Wagih El Masri, Hisatoshi Baba

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


Spinal cord injury is a complex pathology often resulting in functional impairment and paralysis. Gene therapy has emerged as a possible solution to the problems of limited neural tissue regeneration through the administration of factors promoting axonal growth, while also offering long-term local delivery of therapeutic molecules at the injury site. Of note, gene therapy is our response to the requirements of neural and glial cells following spinal cord injury, providing, in a time-dependent manner, growth substances for axonal regeneration and eliminating axonal growth inhibitors. Herein, we explore different gene therapy strategies, including targeting gene expression to modulate the presence of neurotrophic growth or survival factors and increase neural tissue plasticity. Special attention is given to describing advances in viral and non-viral gene delivery systems, as well as the available routes of gene delivery. Finally, we discuss the future of combinatorial gene therapies and give consideration to the implementation of gene therapy in humans.

Original languageEnglish
Pages (from-to)591-607
Number of pages17
JournalTherapeutic Delivery
Issue number5
Publication statusPublished - 2014


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