Creating Patient-Specific Neural Cells for the in Vitro Study of Brain Disorders

Kristen J. Brennand, M. Carol Marchetto, Nissim Benvenisty, Oliver Brüstle, Allison Ebert, Juan Carlos Izpisua Belmonte, Ajamete Kaykas, Madeline A. Lancaster, Frederick J. Livesey, Michael J. McConnell, Ronald D. McKay, Eric M. Morrow, Alysson R. Muotri, David M. Panchision, Lee L. Rubin, Akira Sawa, Frank Soldner, Hongjun Song, Lorenz Studer, Sally TempleFlora M. Vaccarino, Jun Wu, Pierre Vanderhaeghen, Fred H. Gage*, Rudolf Jaenisch

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

66 Scopus citations


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.

Original languageAmerican English
Pages (from-to)933-945
Number of pages13
JournalStem Cell Reports
Issue number6
StatePublished - 8 Dec 2015

Bibliographical note

Funding Information:
This work was supported by the National Institutes of Health through grants R01MH101454 (K.J.B.), R01MH095741 (F.H.G.), U01MH1068821 (F.H.G.), R01NS088538 (R.J.), 2R01MH104610 (R.J.), R01MH102418 (E.M.M.), R01MH105442 (E.M.M.), R01MH094753 (A.R.M.), R01MH103134 (A.R.M.), P01NS066888 (L.L.R.), P50MH094268 (A.S.), R01MH092443 (A.S.), R01MH105660 (A.S.), R33MH087874 (H.S.), U19MH107367 (A.R.M.), R01NS047344 (H.S.), RF1AG042932 (S.T.), R01MH089176 (F.M.V.), and R21MH087879 (F.M.V.); the Department of Defense through award W81XWH-13-1-0414 (F.H.G., M.J.M.); the California Institute for Regenerative Medicine (CIRM) through grant TR4-06747 (A.R.M.); the State of Connecticut (F.M.V.); Maryland Stem Cell Research Fund (H.S.); the Medical Research Council through grant MC_UP_1201/9 (M.A.L.); The Wellcome Trust (Investigator Award to F.J.L.); EU 7th Framework Programme NeuroStemCellRepair through grant 602278 (O.B.); the German Federal Ministry of Education and Research through grants 1615608A (Neuroallianz) (O.B.) and 01ZX1314A (IntegraMent) (O.B.); the North Rhine Westphalian Ministry of Innovation through grant 1403ts007e (StemCellFactory; ) (O.B.); the Belgian FNRS (P.V.); FMRE (P.V.), IUAP (P.V.); WELBIO (P.V.); the ERC (P.V.); the Universidad Católica San Antonio de Murcia (J.C.I.B.); the Brain and Behavior Research Foundation (K.J.B., A.S., A.R.M., F.M.V.); Stanley Foundation (A.S.); the Simons Foundation (F.M.V.); the New York Stem Cell Foundation (K.J.B.); the Harvard Stem Cell Institute (L.L.R.); the Azrieli Foundation (N.B.); SMA Foundation (L.L.R.); S-R/RUSK (A.S.); Regenerative Research Foundation (S.T.); the G. Harold and Leila Y. Mathers Charitable Foundation (J.C.I.B., F.H.G.); The Leona M. and Harry B. Helmsley Charitable Trust (J.C.I.B., F.H.G.); the Engman Foundation (F.H.G.); the JPB Foundation (F.H.G.); Annette C. Merle-Smith (F.H.G.); JPB Foundation (F.H.G.); and Takeda Pharmaceutical International (F.H.G.).

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