title: Creating Patient-Specific Neural Cells for the In Vitro Study of Brain Disorders.
abstract: As a group, we met to discuss the current challenges for creating meaningful patient-specific in vitro models to study brain disorders. Although the convergence of findings between laboratories and patient cohorts provided us confidence and optimism that hiPSC-based platforms will inform future drug discovery efforts, a number of critical technical challenges remain. This opinion piece outlines our collective views on the current state of hiPSC-based disease modeling and discusses what we see to be the critical objectives that must be addressed collectively as a field.
authors.affiliation: Department of Psychiatry, Icahn School of Medicine at Mount Sinai, 1425 Madison Avenue, New York, NY 10029, USA.
authors.forename: Kristen J
authors.initials: KJ
authors.lastname: Brennand
authors.affiliation: Laboratory of Genetics, Salk Institute for Biological Studies, 10010 North Torrey Pines Road, San Diego, CA 92037, USA.
authors.forename: M Carol
authors.initials: MC
authors.lastname: Marchetto
authors.affiliation: Department of Genetics, Hebrew University of Jerusalem, Jerusalem 91904, Israel.
authors.forename: Nissim
authors.initials: N
authors.lastname: Benvenisty
authors.affiliation: Institute of Reconstructive Neurobiology, LIFE & BRAIN Center, University of Bonn, Bonn 53127, Germany.
authors.forename: Oliver
authors.initials: O
authors.lastname: Brüstle
authors.affiliation: Department of Cell Biology, Neurobiology & Anatomy, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI 53226, USA.
authors.forename: Allison
authors.initials: A
authors.lastname: Ebert
authors.affiliation: Gene Expression Laboratory, Salk Institute for Biological Studies, 10010 North Torrey Pines Road, San Diego, CA 92037, USA.
authors.forename: Juan Carlos
authors.initials: JC
authors.lastname: Izpisua Belmonte
authors.affiliation: Department of Neurobiology, Novartis Institute for BioMedical Research, Inc., 100 Technology Square, Cambridge, MA 02139, USA.
authors.forename: Ajamete
authors.initials: A
authors.lastname: Kaykas
authors.affiliation: Laboratory of Molecular Biology, Francis Crick Avenue, Cambridge Biomedical Campus, Cambridge CB2 0QH, UK.
authors.forename: Madeline A
authors.initials: MA
authors.lastname: Lancaster
authors.affiliation: Gurdon Institute, University of Cambridge, Tennis Court Road, Cambridge CB2 1QN, UK.
authors.forename: Frederick J
authors.initials: FJ
authors.lastname: Livesey
authors.affiliation: Department of Biochemistry and Molecular Genetics, Center for Brain Immunology and Glia, University of Virginia School of Medicine, 1340 Jefferson Park Avenue, Charlottesville, VA 22908, USA.
authors.forename: Michael J
authors.initials: MJ
authors.lastname: McConnell
authors.affiliation: Brain Development, Lieber Institute for Brain Development, 855 North Wolfe Street, Baltimore, MD 21205, USA.
authors.forename: Ronald D
authors.initials: RD
authors.lastname: McKay
authors.affiliation: Department of Molecular Biology, Cell Biology and Biochemistry, Brown University, 70 Ship Street, Providence, RI 02912, USA.
authors.forename: Eric M
authors.initials: EM
authors.lastname: Morrow
authors.affiliation: Department of Cellular & Molecular Medicine, University of California, San Diego, 9500 Gilman Drive, La Jolla, CA 92037, USA.
authors.forename: Alysson R
authors.initials: AR
authors.lastname: Muotri
authors.affiliation: Developmental Neurobiology Program, National Institute of Mental Health, 6001 Executive Boulevard, Bethesda, MD 20892, USA.
authors.forename: David M
authors.initials: DM
authors.lastname: Panchision
authors.affiliation: Department of Stem Cell and Regenerative Biology, Harvard University, 16 Divinity Avenue, Cambridge, MA 02138, USA.
authors.forename: Lee L
authors.initials: LL
authors.lastname: Rubin
authors.affiliation: Department of Psychiatry and Behavioral Sciences, The Johns Hopkins Hospital, 600 North Wolfe Street, Baltimore, MD 21287, USA.
authors.forename: Akira
authors.initials: A
authors.lastname: Sawa
authors.affiliation: Whitehead Institute for Biomedical Research, 9 Cambridge Center, Cambridge, MA 02142, USA.
authors.forename: Frank
authors.initials: F
authors.lastname: Soldner
authors.affiliation: Institute for Cell Engineering, Johns Hopkins University School of Medicine, 733 North Broadway, Baltimore, MD 21205, USA.
authors.forename: Hongjun
authors.initials: H
authors.lastname: Song
authors.affiliation: Stem Cell and Tumor Biology, Memorial Sloan-Kettering Cancer Center, 1275 York Avenue, Box 256, New York, NY 10021, USA.
authors.forename: Lorenz
authors.initials: L
authors.lastname: Studer
authors.affiliation: Neural Stem Cell Institute, One Discovery Drive, Rensselaer, NY 12144, USA.
authors.forename: Sally
authors.initials: S
authors.lastname: Temple
authors.affiliation: Child Study Center, Yale University, 230 South Frontage Road, New Haven, CT 06520, USA.
authors.forename: Flora M
authors.initials: FM
authors.lastname: Vaccarino
authors.affiliation: Gene Expression Laboratory, Salk Institute for Biological Studies, 10010 North Torrey Pines Road, San Diego, CA 92037, USA.
authors.forename: Jun
authors.initials: J
authors.lastname: Wu
authors.affiliation: Institute of Interdisciplinary Research (IRIBHM), University of Brussels ULB, 808, Route de Lennik, 1070 Brussels, Belgium.
authors.forename: Pierre
authors.initials: P
authors.lastname: Vanderhaeghen
authors.affiliation: Laboratory of Genetics, Salk Institute for Biological Studies, 10010 North Torrey Pines Road, San Diego, CA 92037, USA. Electronic address: fgage@salk.edu.
authors.forename: Fred H
authors.initials: FH
authors.lastname: Gage
authors.affiliation: Whitehead Institute for Biomedical Research, 9 Cambridge Center, Cambridge, MA 02142, USA. Electronic address: jaenisch@wi.mit.edu.
authors.forename: Rudolf
authors.initials: R
authors.lastname: Jaenisch
mesh_terms: D001921:Brain / Q000187:drug effects / Q000378:metabolism / Q000473:pathology* / Q000503:physiopathology; D001927:Brain Diseases / Q000188:drug therapy / Q000235:genetics / Q000473:pathology* / Q000503:physiopathology; D055808:Drug Discovery / Q000379:methods; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology / Q000378:metabolism / Q000473:pathology*; D009030:Mosaicism; D055495:Neurogenesis; D057285:Precision Medicine / Q000379:methods