Abstract
In this article, Karch, Temple and colleagues describe a resource of fibroblasts, patient-derived induced pluripotent stem cells, and genome engineered stem cells with comprehensive clinical histories that can be accessed by the scientific community for disease modeling and development of novel therapeutics for primary tauopathies.
Original language | English |
---|---|
Pages (from-to) | 939-955 |
Number of pages | 17 |
Journal | Stem Cell Reports |
Volume | 13 |
Issue number | 5 |
DOIs | |
State | Published - 12 Nov 2019 |
Keywords
- CRISPR/Cas9
- MAPT
- corticobasal degeneration
- fibroblasts
- frontotemporal dementia
- induced pluripotent stem cells
- neural progenitor cells
- progressive supranuclear palsy
- tau
- tauopathy
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In: Stem Cell Reports, Vol. 13, No. 5, 12.11.2019, p. 939-955.
Research output: Contribution to journal › Article › peer-review
TY - JOUR
T1 - A Comprehensive Resource for Induced Pluripotent Stem Cells from Patients with Primary Tauopathies
AU - Tau Consortium Stem Cell Group
AU - Karch, Celeste M.
AU - Kao, Aimee W.
AU - Karydas, Anna
AU - Onanuga, Khadijah
AU - Martinez, Rita
AU - Argouarch, Andrea
AU - Wang, Chao
AU - Huang, Cindy
AU - Sohn, Peter Dongmin
AU - Bowles, Kathryn R.
AU - Spina, Salvatore
AU - Silva, M. Catarina
AU - Marsh, Jacob A.
AU - Hsu, Simon
AU - Pugh, Derian A.
AU - Ghoshal, Nupur
AU - Norton, Joanne
AU - Huang, Yadong
AU - Lee, Suzee E.
AU - Seeley, William W.
AU - Theofilas, Panagiotis
AU - Grinberg, Lea T.
AU - Moreno, Fermin
AU - McIlroy, Kathryn
AU - Boeve, Bradley F.
AU - Cairns, Nigel J.
AU - Crary, John F.
AU - Haggarty, Stephen J.
AU - Ichida, Justin K.
AU - Kosik, Kenneth S.
AU - Miller, Bruce L.
AU - Gan, Li
AU - Goate, Alison M.
AU - Temple, Sally
AU - Alquezar, Carolina
AU - Bowles, Kathryn
AU - Butler, David
AU - Hernandez, Israel
AU - Hennes, Valerie
AU - Kampmann, Martin
N1 - Funding Information: We would like to thank the research subjects and their families who generously participated in this study. We thank Steven Lotz, Shawn Sutton, Brian Unruh, Isabel Tian, and Nicholas St. John at NeuraCell and Evan Y. Snyder and Yang M. Lui at Sanford Burnham Prebys Medical Discovery Institute for technical assistance. We thank the Icahn School of Medicine at Mount Sinai Pluripotent Stem Cell Core facility for technical assistance. The gRNAs were generated by the Genome Engineering and iPSC Center (GEiC) at the Washington University in St. Louis. We thank Amber Neilson at the GEiC for technical assistance. This work was supported by access to equipment made possible by the Hope Center for Neurological Disorders , and the Departments of Neurology and Psychiatry at Washington University School of Medicine . Data collection and dissemination of the data presented in this paper were supported by the LEFFTDS & ARTFL Consortium (LEFFTDS: U01AG045390 ; ARTFL: U54NS092089 ). The authors acknowledge the invaluable contributions of the participants in ARTFL & LEFFTDS as well as the assistance of the support staff at each of the participating sites. Funding: The Tau Consortium (C.M.K., A.W.K., Y.H., S.E.L., J.F.C., S.J.H., J.K.I., K.S.K., B.L.M., L.G., A.M.G., S.T., K.O., M.K.), NIH AG046374 (C.M.K.), CurePSP (A.W.K., K.R.B.), Brain Research Foundation (A.W.K.), MGH Research Scholars Program (S.J.H.), Association for Frontotemporal Degeneration , AFTD (M.C.S., K.R.B.), BrightFocus Foundation (K.R.B.), Farrell Family Alzheimer’s Disease Research Fund (C.M.K.), NIH /NIA ( P50 AG023501 , P01 AG019724 , T32 AG023481-11S1 , and P50 AG1657303 to B.L.M.), NIH ( R01AG054008 and R01NS095252 to J.F.C.), NIH ( P50 AG005681 to J.C.M.), NIH ( K12 HD001459 to N.G.), NIH/ NINDS ( R35 NS097277 to S.T.), NIH /NIA ( AG056293 to S.T.), NIH ( K08 AG052648 to S.S.), NIH ( U01 AG045390 to B.B.), NIH U54 NS092089 , U24 AG21886 . Neuracell received support from the Empire State Stem Cell Fund through New York State Department of Health contract # C029158 . Opinions expressed here are solely those of the authors and do not necessarily reflect those of the Empire State Stem Cell Board, the New York State Department of Health, or the State of New York. J.K.I. is a New York Stem Cell Foundation-Robertson Investigator. A.W.K. and M.K. are Paul G. Allen Family Foundation Distinguished Investigators. M.K. is a Chan Zuckerberg Biohub Investigator. A.M.G. is a member of the Scientific Advisory Board for Denali Therapeutics and on the Genetic Scientific Advisory Panel for Pfizer. N.J.C. is a member of the Modeling Alliance of Systems Pharmacology in Tauopathies Scientific Advisory Board. S.J.H. is a member of the Scientific Advisory Board for Rodin Therapeutics, Frequency Therapeutics, and Psy Therapeutics and Souvien Therapeutics, none of whom had involvement in the present study. N.G. has participated or is currently participating in clinical trials of anti-dementia drugs sponsored by Bristol Myers Squibb , Eli Lilly /Avid Radiopharmaceuticals, Janssen Immunotherapy , Novartis , Pfizer , Wyeth , SNIFF (The Study of Nasal Insulin to Fight Forgetfulness) study, and A4 (The Anti-Amyloid Treatment in Asymptomatic Alzheimer's Disease) trial. S.T. is president of StemCultures, scientific co-founder of Luxa Biotech and has served on the scientific advisory boards of Sana Biotechnology and Blue Rock Therapeutics and as consultant to Merck. The remaining authors declare no competing interests. Funding Information: We would like to thank the research subjects and their families who generously participated in this study. We thank Steven Lotz, Shawn Sutton, Brian Unruh, Isabel Tian, and Nicholas St. John at NeuraCell and Evan Y. Snyder and Yang M. Lui at Sanford Burnham Prebys Medical Discovery Institute for technical assistance. We thank the Icahn School of Medicine at Mount Sinai Pluripotent Stem Cell Core facility for technical assistance. The gRNAs were generated by the Genome Engineering and iPSC Center (GEiC) at the Washington University in St. Louis. We thank Amber Neilson at the GEiC for technical assistance. This work was supported by access to equipment made possible by the Hope Center for Neurological Disorders, and the Departments of Neurology and Psychiatry at Washington University School of Medicine. Data collection and dissemination of the data presented in this paper were supported by the LEFFTDS & ARTFL Consortium (LEFFTDS: U01AG045390; ARTFL: U54NS092089). The authors acknowledge the invaluable contributions of the participants in ARTFL & LEFFTDS as well as the assistance of the support staff at each of the participating sites. Funding: The Tau Consortium (C.M.K. A.W.K. Y.H. S.E.L. J.F.C. S.J.H. J.K.I. K.S.K. B.L.M. L.G. A.M.G. S.T. K.O. M.K.), NIH AG046374 (C.M.K.), CurePSP (A.W.K. K.R.B.), Brain Research Foundation (A.W.K.), MGH Research Scholars Program (S.J.H.), Association for Frontotemporal Degeneration, AFTD (M.C.S. K.R.B.), BrightFocus Foundation (K.R.B.), Farrell Family Alzheimer's Disease Research Fund (C.M.K.), NIH/NIA (P50 AG023501, P01 AG019724, T32 AG023481-11S1, and P50 AG1657303 to B.L.M.), NIH (R01AG054008 and R01NS095252 to J.F.C.), NIH (P50 AG005681 to J.C.M.), NIH (K12 HD001459 to N.G.), NIH/NINDS (R35 NS097277 to S.T.), NIH/NIA (AG056293 to S.T.), NIH (K08 AG052648 to S.S.), NIH (U01 AG045390 to B.B.), NIH U54 NS092089, U24 AG21886. Neuracell received support from the Empire State Stem Cell Fund through New York State Department of Health contract #C029158. Opinions expressed here are solely those of the authors and do not necessarily reflect those of the Empire State Stem Cell Board, the New York State Department of Health, or the State of New York. J.K.I. is a New York Stem Cell Foundation-Robertson Investigator. A.W.K. and M.K. are Paul G. Allen Family Foundation Distinguished Investigators. M.K. is a Chan Zuckerberg Biohub Investigator. A.M.G. is a member of the Scientific Advisory Board for Denali Therapeutics and on the Genetic Scientific Advisory Panel for Pfizer. N.J.C. is a member of the Modeling Alliance of Systems Pharmacology in Tauopathies Scientific Advisory Board. S.J.H. is a member of the Scientific Advisory Board for Rodin Therapeutics, Frequency Therapeutics, and Psy Therapeutics and Souvien Therapeutics, none of whom had involvement in the present study. N.G. has participated or is currently participating in clinical trials of anti-dementia drugs sponsored by Bristol Myers Squibb, Eli Lilly/Avid Radiopharmaceuticals, Janssen Immunotherapy, Novartis, Pfizer, Wyeth, SNIFF (The Study of Nasal Insulin to Fight Forgetfulness) study, and A4 (The Anti-Amyloid Treatment in Asymptomatic Alzheimer's Disease) trial. S.T. is president of StemCultures, scientific co-founder of Luxa Biotech and has served on the scientific advisory boards of Sana Biotechnology and Blue Rock Therapeutics and as consultant to Merck. The remaining authors declare no competing interests. Publisher Copyright: © 2019 The Authors
PY - 2019/11/12
Y1 - 2019/11/12
N2 - In this article, Karch, Temple and colleagues describe a resource of fibroblasts, patient-derived induced pluripotent stem cells, and genome engineered stem cells with comprehensive clinical histories that can be accessed by the scientific community for disease modeling and development of novel therapeutics for primary tauopathies.
AB - In this article, Karch, Temple and colleagues describe a resource of fibroblasts, patient-derived induced pluripotent stem cells, and genome engineered stem cells with comprehensive clinical histories that can be accessed by the scientific community for disease modeling and development of novel therapeutics for primary tauopathies.
KW - CRISPR/Cas9
KW - MAPT
KW - corticobasal degeneration
KW - fibroblasts
KW - frontotemporal dementia
KW - induced pluripotent stem cells
KW - neural progenitor cells
KW - progressive supranuclear palsy
KW - tau
KW - tauopathy
UR - http://www.scopus.com/inward/record.url?scp=85074595762&partnerID=8YFLogxK
U2 - 10.1016/j.stemcr.2019.09.006
DO - 10.1016/j.stemcr.2019.09.006
M3 - Article
C2 - 31631020
AN - SCOPUS:85074595762
SN - 2213-6711
VL - 13
SP - 939
EP - 955
JO - Stem Cell Reports
JF - Stem Cell Reports
IS - 5
ER -