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Cited by 17Pluripotent Stem Cells ResearchNeurogenesis and neuroplasticity mechanismsDevelopmental Biology and Gene Regulation

Human organoids to model the developing human neocortex in health and disease

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Khakipoor, Shokoufeh, Crouch, Elizabeth E., Mayer, Simone·2020·Brain Research
DOI 10.1016/j.brainres.2020.146803PMC PMC7352040
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Abstract

Rodent models have catalyzed major discoveries in the neocortex, a brain region unique to mammals. However, since the neocortex has expanded considerably in primates, employing rodent models has limitations. Human fetal brain tissue is a scarce resource with limitations for experimental manipulations. In order to create an experimentally tractable representation of human brain development, a number of labs have recently created in vitro models of the developing human brain. These models, generated using human embryonic stem cells or induced pluripotent stem cells, are called "organoids". Organoids have successfully and rapidly uncovered new mechanisms of human brain development in health and disease. In the future, we envision that this strategy will enable faster and more efficient translation of basic neuroscience findings to therapeutic applications. In this review, we discuss the generation of the first human cerebral organoids, progress since their debut, and challenges to be overcome in the future.

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  • Cerebral organoids derived from AD patient-specific iPSCs can recapture patient-specific phenotypes and hence serve as excellent disease models for AD research and drug development.

    “Cerebral organoids derived from AD patient-specific iPSCs can recapture patient-specific phenotypes and hence serve as excellent disease models for AD research and drug development [51–53].”

    From CI-994 is a dual modulator of class I HDACs and Wnt/β-catenin signaling for the treatment of Alzheimer's disease (2026) · cites this paper as [52]

    The Future perspectives section reports that forebrain organoids modeling Alzheimer’s disease mimic amyloid and tau pathology and respond to drug treatment. It does not specify that those organoids came from AD patient-specific iPSCs or recaptured patient-specific phenotypes; it describes organoid-based personalized treatment testing as a future possibility, not a current capability. Page numbers are unavailable in our copy. (LLM, conf=0.87)

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Cited by

Sorted by citations

Authors
Khakipoor, S., Crouch, E. E., & Mayer, S.
Affiliations
  • Hertie Institute for Clinical Brain Research (DE)
  • University of California, San Francisco (US)
Corresponding author
Mayer, Simone
ORCID
Venue
Brain Research
Publisher
Elsevier BV
Published
2020-09
Volume
1742
Pages
146803
ISSN
0006-8993 (ISSN-L)
DOI
10.1016/j.brainres.2020.146803
PMC
PMC7352040
OpenAlex
W3013165669
Cited by
17
References
24
Citations by year
2026: 3 · 2025: 1 · 2024: 3 · 2023: 3 · 2022: 3 · 2021: 3 · 2020: 1
FWCI
0.24
Type
journal article
Language
en
Open access
Licence
Locations
  • Brain Research (publishedVersion, cc-by-nc-nd)
  • PubMed (publishedVersion)
  • PubMed Central (acceptedVersion)
  • eScholarship (California Digital Library) (submittedVersion, cc-by-nc-nd)
Funders
National Institutes of Health
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Pluripotent Stem Cells Research
  • Life Sciences › Neuroscience › Developmental Neuroscience › Neurogenesis and neuroplasticity mechanisms
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Developmental Biology and Gene Regulation
Keywords
brain organoids, neocortex development, human brain development, human embryonic stem cells, induced pluripotent stem cells, in vitro brain models, neurodevelopmental disorders, translational neuroscience
MeSH terms
Brain, Humans, Models, Biological, Organoids, Cell Culture Techniques, Neocortex, Neurogenesis, Induced Pluripotent Stem Cells

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