Preclinical studies for induced pluripotent stem cell-based therapeutics [Cell Biology]

December 20th, 2013 by Harding, J., Mirochnitchenko, O.

Induced Pluripotent Stem Cells (iPSCs) and their differentiated derivatives can potentially be applied to cell-based therapy of human diseases. The properties of iPSCs are being studied intensively, both to understand the basic biology of pluripotency and cellular differentiation as well as to solve problems associated with therapeutic applications. Examples of specific preclinical applications summarized briefly in this review include use of iPSCs to treat diseases of the liver, nervous system, eye and heart and metabolic conditions such as diabetes. Early stage studies illustrate the potential of iPSC-derived cells and have identified several challenges that must be addressed before moving to clinical trials. These include rigorous quality control and efficient production of required cell populations, improvement of cell survival and engraftment and development of technologies to monitor transplanted cell behavior for extended periods of time. Problems related to immune rejection, genetic instability and tumorigenicity must be solved. Testing the efficacy of iPSC-based therapies requires further improvement of animal models precisely recapitulating human disease conditions.