title: New lessons learned from disease modeling with induced pluripotent stem cells.
abstract: Cellular reprogramming and generation of induced pluripotent stem cells (iPSCs) from adult cell types have enabled the creation of patient-specific stem cells for use in disease modeling. To date, many iPSC lines have been generated from a variety of disorders, which have then been differentiated into disease-relevant cell types. When a disease-specific phenotype is detectable in such differentiated cells, the reprogramming technology provides a new opportunity to identify aberrant disease-associated pathways and drugs that can block them. Here, we highlight recent progress as well as limitations in the use of iPSCs to recapitulate disease phenotypes and to screen for therapeutics in vitro.
authors: Stem Cell Transplantation Program, Division of Pediatric Hematology and Oncology, Manton Center for Orphan Disease Research, Howard Hughes Medical Institute, Children's Hospital Boston and Dana Farber Cancer Institute, Boston, MA, USA.
authors: Tamer T
authors: TT
authors: Onder
authors: George Q
authors: GQ
authors: Daley
mesh_terms: D002454:Cell Differentiation; D002478:Cells, Cultured; D065150:Cellular Reprogramming; D055808:Drug Discovery / Q000379:methods*; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology* / Q000378:metabolism / Q000473:pathology; D008954:Models, Biological; D010641:Phenotype