Human iPSC for Therapeutic Approaches to the Nervous System: Present and Future Applications

Many central nervous system (CNS) diseases including stroke, spinal cord injury (SCI), and brain tumors are a significant cause ofworldwide morbidity/mortality and yet do not have satisfying treatments. Cell-based therapy to restore lost function or to carry newtherapeutic genes is a promising new therapeutic approach, particularly after human iPSCs became available. However, efficientgeneration of footprint-free and xeno-free human iPSC is a prerequisite for their clinical use. In this paper, we will first summarizethe current methodology to obtain footprint- and xeno-free human iPSC. We will then review the current iPSC applications intherapeutic approaches for CNS regeneration and their use as vectors to carry proapoptotic genes for brain tumors and reviewtheir applications for modelling of neurological diseases and formulating new therapeutic approaches. Available results will besummarized and compared. Finally, we will discuss current limitations precluding iPSC from being used on large scale for clinicalapplications and provide an overview of future areas of improvement. In conclusion, significant progress has occurred in derivingiPSC suitable for clinical use in the field of neurological diseases. Current efforts to overcome technical challenges, includingreducing labour and cost, will hopefully expedite the integration of this technology in the clinical setting