Evidence map›Paper›PMID 30875083›Full record

ReviewAnnals of the New York Academy of Sciences2020

Human pluripotent stem cell-derived models and drug screening in CNS precision medicine.

M Catarina Silva, Stephen J Haggarty

Abstract readReview
In one paragraph

Review in Annals of the New York Academy of Sciences, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 54 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
54citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

54 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Mental health dished up-the use of iPSC models in neuropsychiatric research.Journal of neural transmission (Vienna, Austria : 1996) · 2020
    Pooled it
  2. Review
  3. Review
  4. Article
  5. Article
  6. Artificial Intelligence Across the Drug Development Lifecycle.Medical sciences (Basel, Switzerland) · 2026
    Review
  7. Review
  8. Functional Neurogenomics to Dissect Disease Mechanisms Across Models.Annual review of genomics and human genetics · 2025
    Review
  9. Review
  10. Review
  11. Personalized Stem Cell-Based Regeneration in Spinal Cord Injury Care.International journal of molecular sciences · 2025
    Review
  12. Article
  13. Article
  14. Article
  15. Article
  16. Review
  17. Review
  18. Breaking the mold: 3D cell cultures reshaping the future of cancer research.Frontiers in cell and developmental biology · 2024
    Review
  19. Review
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

2 authors.

M Catarina SilvaChemical Neurobiology Laboratory, Departments of Neurology and Psychiatry, Massachusetts General Hospital, Center for Genomic Medicine, Harvard Medical School, Boston, Massachusetts.ORCID 0000-0001-5421-6673
Stephen J HaggartyChemical Neurobiology Laboratory, Departments of Neurology and Psychiatry, Massachusetts General Hospital, Center for Genomic Medicine, Harvard Medical School, Boston, Massachusetts.

Funding

Research ProjectP50MH106933 · NIMH · HARVARD MEDICAL SCHOOL · PI KOHANE, ISAAC S. · 2014 to 2018
$17.0M
Validation of Modulators of PGRN as Novel Therapeutics for Frontotemporal DementiR01NS108115 · NINDS · UT SOUTHWESTERN MEDICAL CENTER · PI HAGGARTY, STEPHEN J, HERZ, JOACHIM J · 2018 to 2022
$4.0M
Chemical Genomic Approaches to Neurobiology of DISC1R01MH091115 · NIMH · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · PI TSAI, LI-HUEI · 2010 to 2014
$3.4M
Functional Genomics of Neuroplasticity in SchizophreniaR01MH095088 · NIMH · BROAD INSTITUTE, INC. · PI HAGGARTY, STEPHEN J · 2011 to 2014
$2.5M
Patient-Derived Cellular Models of Putative AntidepressantsR01AT009144 · NCCIH · MASSACHUSETTS GENERAL HOSPITAL · PI HAGGARTY, STEPHEN J, PERLIS, ROY H. · 2015 to 2019
$2.0M
Genomics-Guided Characterization of iPS Cells from Common Mental IllnessesR33MH087896 · NIMH · MASSACHUSETTS GENERAL HOSPITAL · PI HAGGARTY, STEPHEN J · 2011 to 2012
$872k
Autosomal Dominant FTLD Tauopathy Patient-Specific Stem Cell ModelsR21NS085487 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI DICKERSON, BRADFORD C, HAGGARTY, STEPHEN J · 2014 to 2015
$479k
Epigenomic Characterization of Alzheimer's Disease Neurons from iPSCsRF1AG042978 · NIA · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · PI HAGGARTY, STEPHEN J, TSAI, LI-HUEI · 2012 to 2012
$429k
NCCIH NIH HHS R01 AT009144NCCIH NIH HHS R01AT009144NHGRI NIH HHS P50MH106933NIA NIH HHS RF1 AG042978NIMH NIH HHS P50 MH106933NIMH NIH HHS R01 MH091115NIMH NIH HHS R01MH091115NIMH NIH HHS R01 MH095088NIMH NIH HHS R01MH095088NIMH NIH HHS R33 MH087896NIMH NIH HHS R33MH087896NINDS NIH HHS R01 NS108115NINDS NIH HHS R01NS108115NINDS NIH HHS R21 NS085487NINDS NIH HHS R21NS085487
6 · The paper itself

Abstract

Development of effective therapeutics for neurological disorders has historically been challenging partly because of lack of accurate model systems in which to investigate disease etiology and test new therapeutics at the preclinical stage. Human stem cells, particularly patient-derived induced pluripotent stem cells (iPSCs) upon differentiation, have the ability to recapitulate aspects of disease pathophysiology and are increasingly recognized as robust scalable systems for drug discovery. We review advances in deriving cellular models of human central nervous system (CNS) disorders using iPSCs along with strategies for investigating disease-relevant phenotypes, translatable biomarkers, and therapeutic targets. Given their potential to identify novel therapeutic targets and leads, we focus on phenotype-based, small-molecule screens employing human stem cell-derived models. Integrated efforts to assemble patient iPSC-derived cell models with deeply annotated clinicopathological data, along with molecular and drug-response signatures, may aid in the stratification of patients, diagnostics, and clinical trial success, shifting translational science and precision medicine approaches. A number of remaining challenges, including the optimization of cost-effective, large-scale culture of iPSC-derived cell types, incorporation of aging into neuronal models, as well as robustness and automation of phenotypic assays to support quantitative drug efficacy, toxicity, and metabolism testing workflows, are covered. Continued advancement of the field is expected to help fully humanize the process of CNS drug discovery.

Indexed as

Drug Evaluation, PreclinicalPrecision MedicineCell DifferentiationCentral Nervous SystemCentral Nervous System DiseasesDrug DiscoveryHumansInduced Pluripotent Stem CellsNeuronsdrug discoveryhuman-induced pluripotent stem cellsneurodegenerative disordersneurosciencepsychiatric disordersscreening

Identifiers

PMID30875083
PMCPMC8193821

What OpenQuestion holds

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Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.