Table 1:

Disease models in human brain organoids.

Disease	Paper	Guided/Unguided system	Region	Main findings	Protocol used
Microcephaly	Lancaster et al 2013 (Nature)	Unguided	Whole-brain organoids	Increase in immature neuronal differentiation	Lancaster et al 2013
Autism spectrum disorder	Mariani et al 2015 (Cell)	Guided	Telencephalon organoids	Disruption of excitatory/inhibitor balance	Mariani et al 2015
ZIKA virus exposure	Qian et al 2016 (Cell)	Guided	Forebrain organoids	Increase in apoptosis and suppressing neuronal progenitor proliferation	Qian et al 2016
Alzheimer disease	Raja et al 2016 (PLOS PNE)	Guided	Forebrain organoids	Amyloid and tau pathology is mimicked and their responsiveness to drug treatment	Adopted from Kadoshima et al 2013 protocol
Lissencephaly (Miller Dieker Syndrome)	Bershteyn et al 2017 (Cell Stem Cell)	Guided	Cerebral organoids	A decrease in radial glial cells population and defect in neuronal migration	Kadoshima et al 2013
Zika virus	Watanabe et al 2017 (Cell Report)	Guided	Cerebral organoids	Increase apoptosis and hence smaller organoids size. Further efficiency of different drug treatment	Adopted from Kadoshima et al 2013 protocol
Schizophrenia	Ye et al 2017 (Neuron)	Guided	Forebrain organoids	Reduced proliferation of neural stem cells in the ventricular zone	Qian et al 2016
Microcephaly	Li et al 2017 (Protein & Cell)	Guided	Telencephalon organoids	A decrease in neuronal progenitors and mature neurons.	Adopted from Lancaster et al 2013 (Li et al 2017)
Frontotemporal dementia	Seo et al 2017 (The Journal of neuroscience)	Guided	Forebrain organoids	The pathway involved in Tau phosphorylation	Adopted from Kadoshima et al 2013 protocol
Brain tumor formation	Bian et al 2018 (Nature method)	Unguided	Cerebral organoids	Targeted drug testing on different brain tumors	Lancaster et al 2013
Periventricular heterotopia	Blair et al 2018 (Nature medicine)	Unguided	Cerebral organoids	Defective neuronal migration, and morphological disturbance in neural progenitor cells	Lancaster et al 2013
Autism spectrum disorder	Schafer et al 2019 (Nature neuroscience)	Guided and Unguided organoids	Cerebral and forebrain organoids	Increase in neurite length of the early born neurons	Lancaster et al 2013; Qian et al 2016
Tuberous sclerosis	Klaus et al 2019 (Nature medicine)	Guided	Cortical spheroids	Disruption of mTORCI signaling pathway during development	Pasca et al 2015
Hypoxic brain injury of prematurity	Pasca et al 2019 (Nature medicine)	Guided	Cortical spheroids	Premature differentiation of intermediate progenitor cells	Pasca et al 2019
Prenatal Hypoxic Injury	Daviaud et al 2019 (frontiers in Cellular Neuroscience)	Unguided	Cerebral organoids	Varying resilience from different neural populations.	Adopted from Lancaster et al 2013