New lessons learned from disease modeling with induced pluripotent stem cells.

Curr Opin Genet Dev

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.

Published: October 2012

AI Article Synopsis

  • Cellular reprogramming allows adult cells to become induced pluripotent stem cells (iPSCs), creating patient-specific stem cells for studying diseases.
  • Many iPSC lines have been developed from various disorders, enabling the differentiation of these cells into types that reflect disease characteristics.
  • This technology offers a way to discover abnormal disease pathways and potential drugs, although there are still challenges in accurately modeling diseases and screening for treatments in the lab.

Article 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.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3489983PMC
http://dx.doi.org/10.1016/j.gde.2012.05.005DOI Listing

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