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 of worldwide morbidity/mortality and yet do not have satisfying treatments. Cell-based therapy to restore lost function or to carry new therapeutic genes is a promising new therapeutic approach, particularly after human iPSCs became available. Significant progress has occurred in deriving iPSC suitable for clinical use in the field of neurological diseases. Current efforts to overcome technical challenges, including reducing labour and cost, will hopefully expedite the integration of this technology in the clinical setting.

• 1
  Ischemic stroke, still causing high disability and mortality, prompted the investigation of therapeutic approaches other than thrombolytic therapy and/or percutaneous intravascular interventions. iPSCs have emerged as a promising tool for cell replacement in ischemic brain injuries.
• 2
  Patient-specific iPSCs provide the unprecedented opportunity to study insights and potentially develop therapeutic options for neurodegenerative diseases, up to date difficult to target due to lack of experimental models.
• 3
  We have shown that we can differentiate astrocyte from iPSC in similar fashion to those obtained from ESC [21]. Recently, we have also shown that we can differentiate a pure population of footprint-free iPSC-derived astrocytes (Figure 3), which does not cause teratogenicity after implant into the brain [53].