title: Concise review: modeling central nervous system diseases using induced pluripotent stem cells.
abstract: Induced pluripotent stem cells (iPSCs) offer an opportunity to delve into the mechanisms underlying development while also affording the potential to take advantage of a number of naturally occurring mutations that contribute to either disease susceptibility or resistance. Just as with any new field, several models of screening are being explored, and innovators are working on the most efficient methods to overcome the inherent limitations of primary cell screens using iPSCs. In the present review, we provide a background regarding why iPSCs represent a paradigm shift for central nervous system (CNS) disease modeling. We describe the efforts in the field to develop more biologically relevant CNS disease models, which should provide screening assays useful for the pharmaceutical industry. We also provide some examples of successful uses for iPSC-based screens and suggest that additional development could revolutionize the field of drug discovery. The development and implementation of these advanced iPSC-based screens will create a more efficient disease-specific process underpinned by the biological mechanism in a patient- and disease-specific manner rather than by trial-and-error. Moreover, with careful and strategic planning, shared resources can be developed that will enable exponential advances in the field. This will undoubtedly lead to more sensitive and accurate screens for early diagnosis and allow the identification of patient-specific therapies, thus, paving the way to personalized medicine.
authors.affiliation: XCell Science Inc., Novato, California, USA; Buck Institute for Research on Aging, Novato, California, USA; Laboratory of Stem Cell Biology, NIH Center for Regenerative Medicine, Bethesda, Maryland, USA; National Center for Advancing Translational Sciences, NIH, Bethesda, Maryland, USA; New York Stem Cell Foundation, New York, New York, USA xzeng@xcellscience.com.
authors.forename: Xianmin
authors.initials: X
authors.lastname: Zeng
authors.affiliation: XCell Science Inc., Novato, California, USA; Buck Institute for Research on Aging, Novato, California, USA; Laboratory of Stem Cell Biology, NIH Center for Regenerative Medicine, Bethesda, Maryland, USA; National Center for Advancing Translational Sciences, NIH, Bethesda, Maryland, USA; New York Stem Cell Foundation, New York, New York, USA.
authors.forename: Joshua G
authors.initials: JG
authors.lastname: Hunsberger
authors.affiliation: XCell Science Inc., Novato, California, USA; Buck Institute for Research on Aging, Novato, California, USA; Laboratory of Stem Cell Biology, NIH Center for Regenerative Medicine, Bethesda, Maryland, USA; National Center for Advancing Translational Sciences, NIH, Bethesda, Maryland, USA; New York Stem Cell Foundation, New York, New York, USA.
authors.forename: Anton
authors.initials: A
authors.lastname: Simeonov
authors.affiliation: XCell Science Inc., Novato, California, USA; Buck Institute for Research on Aging, Novato, California, USA; Laboratory of Stem Cell Biology, NIH Center for Regenerative Medicine, Bethesda, Maryland, USA; National Center for Advancing Translational Sciences, NIH, Bethesda, Maryland, USA; New York Stem Cell Foundation, New York, New York, USA.
authors.forename: Nasir
authors.initials: N
authors.lastname: Malik
authors.affiliation: XCell Science Inc., Novato, California, USA; Buck Institute for Research on Aging, Novato, California, USA; Laboratory of Stem Cell Biology, NIH Center for Regenerative Medicine, Bethesda, Maryland, USA; National Center for Advancing Translational Sciences, NIH, Bethesda, Maryland, USA; New York Stem Cell Foundation, New York, New York, USA.
authors.forename: Ying
authors.initials: Y
authors.lastname: Pei
authors.affiliation: XCell Science Inc., Novato, California, USA; Buck Institute for Research on Aging, Novato, California, USA; Laboratory of Stem Cell Biology, NIH Center for Regenerative Medicine, Bethesda, Maryland, USA; National Center for Advancing Translational Sciences, NIH, Bethesda, Maryland, USA; New York Stem Cell Foundation, New York, New York, USA.
authors.forename: Mahendra
authors.initials: M
authors.lastname: Rao
mesh_terms: D002493:Central Nervous System Diseases / Q000188:drug therapy*; D055808:Drug Discovery / Q000379:methods*; D006801:Humans; D057026:Induced Pluripotent Stem Cells; D008959:Models, Neurological; D057285:Precision Medicine / Q000379:methods
keywords: Cell-based screening; Central nervous system disease; Genetic engineering; iPSC