Abstract
The central nervous system (CNS) has very limited potential for regeneration and repair so many neurological disorders can cause permanent functional sequelae. One strategy to improve functional recovery is to use exogenous supplementation of stem cells. Therapeutic effects of stem cells are being segmented using new gene therapy technologies. Stem cell-based gene therapy is a novel technology that maximizes the therapeutic effects of stem cells by engineering them to express therapeutic genes specific to the pathophysiology of the target disorder. This method combines the advantages of stem cell and gene therapy together: Regeneration of functional neural tissue by stem cells and modulation of pathophysiology by therapeutic genes. Many current studies are aimed at overcoming the obstacles to stem cell-based gene therapy and moving these novel treatments into the clinic. Here, we review the current status of stem cell-based gene therapy against neurological diseases and discuss critical issues in the development of stem cell-based gene therapy, including both technical and regulatory issues.
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