title: Preclinical studies for induced pluripotent stem cell-based therapeutics.
abstract: Induced pluripotent stem cells (iPSCs) and their differentiated derivatives can potentially be applied to cell-based therapy for human diseases. The properties of iPSCs are being studied intensively both to understand the basic biology of pluripotency and cellular differentiation and to solve problems associated with therapeutic applications. Examples of specific preclinical applications summarized briefly in this minireview include the 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.
authors.affiliation: From the Division of Comparative Medicine, Office of Research Infrastructure Programs, Division of Program Coordination, Planning, and Strategic Initiatives, Office of the Director, National Institutes of Health, Bethesda, Maryland 20892.
authors.forename: John
authors.initials: J
authors.lastname: Harding
authors.forename: Oleg
authors.initials: O
authors.lastname: Mirochnitchenko
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology* / Q000637:transplantation*; D033581:Stem Cell Transplantation
keywords: Animal Models; Differentiation; Induced Pluripotent Stem Cell (iPSC); Regenerative Medicine; Stem Cells