title: A practical guide to induced pluripotent stem cell research using patient samples.
abstract: Approximately 3 years ago, we assessed how patient induced pluripotent stem cell (iPSC) research could potentially impact human pathobiology studies in the future. Since then, the field has grown considerably with numerous technical developments, and the idea of modeling diseases 'in a dish' is becoming increasingly popular in biomedical research. Likely, it is even acceptable to include patient iPSCs as one of the standard research tools for disease mechanism studies, just like knockout mice. However, as the field matures, we acknowledge there remain many practical limitations and obstacles for their genuine application to understand diseases, and accept that it has not been as straightforward to model disorders as initially proposed. A major practical challenge has been efficient direction of iPSC differentiation into desired lineages and preparation of the large numbers of specific cell types required for study. Another even larger obstacle is the limited value of in vitro outcomes, which often do not closely represent disease conditions. To overcome the latter issue, many new approaches are underway, including three-dimensional organoid cultures from iPSCs, xenotransplantation of human cells to animal models and in vitro interaction of multiple cell types derived from isogenic iPSCs. Here we summarize the areas where patient iPSC studies have provided truly valuable information beyond existing skepticism, discuss the desired technologies to overcome current limitations and include practical guidance for how to utilize the resources. Undoubtedly, these human patient cells are an asset for experimental pathology studies. The future rests on how wisely we use them.
authors.affiliation: Department of Pathology, Immunology and Laboratory Medicine, Center for Cellular Reprogramming, University of Florida College of Medicine, Gainesville, FL, USA.
authors.forename: Katherine E
authors.initials: KE
authors.lastname: Santostefano
authors.affiliation: Department of Molecular Genetics and Microbiology, Center for Cellular Reprogramming, University of Florida College of Medicine, Gainesville, FL, USA.
authors.forename: Takashi
authors.initials: T
authors.lastname: Hamazaki
authors.affiliation: Department of Pathology, Immunology and Laboratory Medicine, Center for Cellular Reprogramming, University of Florida College of Medicine, Gainesville, FL, USA.
authors.forename: Nikolett M
authors.initials: NM
authors.lastname: Biel
authors.affiliation: Department of Pediatrics, Osaka City University, Osaka, Japan.
authors.forename: Shouguang
authors.initials: S
authors.lastname: Jin
authors.affiliation: Department of Reproductive Biology and Pathology, National Research Institute for Child Health and Development, Tokyo, Japan.
authors.forename: Akihiro
authors.initials: A
authors.lastname: Umezawa
authors.affiliation: Department of Pathology, Immunology and Laboratory Medicine, Center for Cellular Reprogramming, University of Florida College of Medicine, Gainesville, FL, USA.
authors.forename: Naohiro
authors.initials: N
authors.lastname: Terada
mesh_terms: D035843:Biomedical Research / Q000379:methods*; D006801:Humans; D039904:Pluripotent Stem Cells / Q000166:cytology*
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