1. Boyle CA, Boulet S, Schieve LA, Cohen RA, Blumberg SJ, Yeargin-Allsopp M, Visser S, Kogan MD. 2011; Trends in the prevalence of developmental disabilities in US children, 1997-2008. Pediatrics. 127:1034–1042. DOI:
10.1542/peds.2010-2989. PMID:
21606152.

2. Thomson JA, Itskovitz-Eldor J, Shapiro SS, Waknitz MA, Swiergiel JJ, Marshall VS, Jones JM. 1998; Embryonic stem cell lines derived from human blastocysts. Science. 282:1145–1147. Erratum in: Science 1998;282:1827. DOI:
10.1126/science.282.5391.1145. PMID:
9804556.

3. Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, Tomoda K, Yamanaka S. 2007; Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell. 131:861–872. DOI:
10.1016/j.cell.2007.11.019. PMID:
18035408.

4. Li L, Chao J, Shi Y. 2018; Modeling neurological diseases using iPSC-derived neural cells: iPSC modeling of neurological diseases. Cell Tissue Res. 371:143–151. DOI:
10.1007/s00441-017-2713-x. PMID:
29079884. PMCID:
PMC6029980.

5. Mariappan I, Maddileti S, Joseph P, Siamwala JH, Vauhini V. 2015; Enriched cultures of retinal cells from BJNhem20 human embryonic stem cell line of Indian origin. Invest Ophthalmol Vis Sci. 56:6714–6723. DOI:
10.1167/iovs.15-17364. PMID:
26567782.

6. Park IH, Arora N, Huo H, Maherali N, Ahfeldt T, Shimamura A, Lensch MW, Cowan C, Hochedlinger K, Daley GQ. 2008; Disease-specific induced pluripotent stem cells. Cell. 134:877–886. DOI:
10.1016/j.cell.2008.07.041. PMID:
18691744. PMCID:
PMC2633781.

7. Eiraku M, Watanabe K, Matsuo-Takasaki M, Kawada M, Yonemura S, Matsumura M, Wataya T, Nishiyama A, Muguruma K, Sasai Y. 2008; Self-organized formation of polarized cortical tissues from ESCs and its active manipulation by extrinsic signals. Cell Stem Cell. 3:519–532. DOI:
10.1016/j.stem.2008.09.002. PMID:
18983967.

8. Lancaster MA, Renner M, Martin CA, Wenzel D, Bicknell LS, Hurles ME, Homfray T, Penninger JM, Jackson AP, Knoblich JA. 2013; Cerebral organoids model human brain development and microcephaly. Nature. 501:373–379. DOI:
10.1038/nature12517. PMID:
23995685. PMCID:
PMC3817409.

9. Camp JG, Badsha F, Florio M, Kanton S, Gerber T, Wilsch-Bräuninger M, Lewitus E, Sykes A, Hevers W, Lancaster M, Knoblich JA, Lachmann R, Pääbo S, Huttner WB, Treutlein B. 2015; Human cerebral organoids recapitulate gene expression programs of fetal neocortex development. Proc Natl Acad Sci U S A. 112:15672–15677. DOI:
10.1073/pnas.1520760112. PMID:
26644564. PMCID:
PMC4697386.

10. Lancaster MA, Corsini NS, Wolfinger S, Gustafson EH, Phillips AW, Burkard TR, Otani T, Livesey FJ, Knoblich JA. 2017; Guided self-organization and cortical plate formation in human brain organoids. Nat Biotechnol. 35:659–666. Erratum in: Nat Biotechnol 2018;36:1016. DOI:
10.1038/nbt.3906. PMID:
28562594. PMCID:
PMC5824977.

11. Quadrato G, Nguyen T, Macosko EZ, Sherwood JL, Min Yang S, Berger DR, Maria N, Scholvin J, Goldman M, Kinney JP, Boyden ES, Lichtman JW, Williams ZM, McCarroll SA, Arlotta P. 2017; Cell diversity and network dynamics in photosensitive human brain organoids. Nature. 545:48–53. DOI:
10.1038/nature22047. PMID:
28445462. PMCID:
PMC5659341.

15. Zhang W, Yang SL, Yang M, Herrlinger S, Shao Q, Collar JL, Fierro E, Shi Y, Liu A, Lu H, Herring BE, Guo ML, Buch S, Zhao Z, Xu J, Lu Z, Chen JF. 2019; Modeling microcephaly with cerebral organoids reveals a WDR62-CEP170-KIF2A pathway promoting cilium disassembly in neural progenitors. Nat Commun. 10:2612. DOI:
10.1038/s41467-019-10497-2. PMID:
31197141. PMCID:
PMC6565620. PMID:
fe809d4eaad64cd5995ce2a720fd1c05.

16. Coulter ME, Dorobantu CM, Lodewijk GA, Delalande F, Cianferani S, Ganesh VS, Smith RS, Lim ET, Xu CS, Pang S, Wong ET, Lidov HGW, Calicchio ML, Yang E, Gonzalez DM, Schlaeger TM, Mochida GH, Hess H, Lee WA, Lehtinen MK, Kirchhausen T, Haussler D, Jacobs FMJ, Gaudin R, Walsh CA. 2018; The ESCRT-III protein CHMP1A mediates secretion of sonic hedgehog on a distinctive subtype of extracellular vesicles. Cell Rep. 24:973–986.e8. DOI:
10.1016/j.celrep.2018.06.100. PMID:
30044992. PMCID:
PMC6178983.

17. Jin M, Pomp O, Shinoda T, Toba S, Torisawa T, Furuta K, Oiwa K, Yasunaga T, Kitagawa D, Matsumura S, Miyata T, Tan TT, Reversade B, Hirotsune S. 2017; Katanin p80, NuMA and cytoplasmic dynein cooperate to control microtubule dynamics. Sci Rep. 7:39902. DOI:
10.1038/srep39902. PMID:
28079116. PMCID:
PMC5228124.

18. Bershteyn M, Nowakowski TJ, Pollen AA, Di Lullo E, Nene A, Wynshaw-Boris A, Kriegstein AR. 2017; Human iPSC-derived cerebral organoids model cellular features of lissencephaly and reveal prolonged mitosis of outer radial glia. Cell Stem Cell. 20:435–449.e4. DOI:
10.1016/j.stem.2016.12.007. PMID:
28111201. PMCID:
PMC5667944.

19. Li Y, Muffat J, Omer A, Bosch I, Lancaster MA, Sur M, Gehrke L, Knoblich JA, Jaenisch R. 2017; Induction of expansion and folding in human cerebral organoids. Cell Stem Cell. 20:385–396.e3. DOI:
10.1016/j.stem.2016.11.017. PMID:
28041895. PMCID:
PMC6461394.

20. Gabriel E, Wason A, Ramani A, Gooi LM, Keller P, Pozniakovsky A, Poser I, Noack F, Telugu NS, Calegari F, Šarić T, Hescheler J, Hyman AA, Gottardo M, Callaini G, Alkuraya FS, Gopalakrishnan J. 2016; CPAP promotes timely cilium disassembly to maintain neural progenitor pool. EMBO J. 35:803–819. DOI:
10.15252/embj.201593679. PMID:
26929011. PMCID:
PMC4972140.

21. O'Neill AC, Kyrousi C, Klaus J, Leventer RJ, Kirk EP, Fry A, Pilz DT, Morgan T, Jenkins ZA, Drukker M, Berkovic SF, Scheffer IE, Guerrini R, Markie DM, Götz M, Cappello S, Robertson SP. 2018; A primate-specific isoform of PLEKHG6 regulates neurogenesis and neuronal migration. Cell Rep. 25:2729–2741.e6. DOI:
10.1016/j.celrep.2018.11.029. PMID:
30517861.
22. Klaus J, Kanton S, Kyrousi C, Ayo-Martin AC, Di Giaimo R, Riesenberg S, O'Neill AC, Camp JG, Tocco C, Santel M, Rusha E, Drukker M, Schroeder M, Götz M, Robertson SP, Treutlein B, Cappello S. 2019; Altered neuronal migratory trajectories in human cerebral organoids derived from individuals with neuronal heterotopia. Nat Med. 25:561–568. DOI:
10.1038/s41591-019-0371-0. PMID:
30858616.

23. Srikanth P, Lagomarsino VN, Muratore CR, Ryu SC, He A, Taylor WM, Zhou C, Arellano M, Young-Pearse TL. 2018; Shared effects of DISC1 disruption and elevated WNT signaling in human cerebral organoids. Transl Psychiatry. 8:77. DOI:
10.1038/s41398-018-0122-x. PMID:
29643329. PMCID:
PMC5895714.

24. Johnstone M, Vasistha NA, Barbu MC, Dando O, Burr K, Christopher E, Glen S, Robert C, Fetit R, Macleod KG, Livesey MR, Clair DS, Blackwood DHR, Millar K, Carragher NO, Hardingham GE, Wyllie DJA, Johnstone EC, Whalley HC, McIntosh AM, Lawrie SM, Chandran S. 2019; Reversal of proliferation deficits caused by chromosome 16p13.11 microduplication through targeting NFκB signaling: an integrated study of patient-derived neuronal precursor cells, cerebral organoids and in vivo brain imaging. Mol Psychiatry. 24:294–311. DOI:
10.1038/s41380-018-0292-1. PMID:
30401811. PMCID:
PMC6344377.

25. Stachowiak EK, Benson CA, Narla ST, Dimitri A, Chuye LEB, Dhiman S, Harikrishnan K, Elahi S, Freedman D, Brennand KJ, Sarder P, Stachowiak MK. 2017; Cerebral organoids reveal early cortical maldevelopment in schizophrenia-computational anatomy and genomics, role of FGFR1. Transl Psychiatry. 7:6. DOI:
10.1038/s41398-017-0054-x. PMID:
30446636. PMCID:
PMC5802550.

26. Wang P, Mokhtari R, Pedrosa E, Kirschenbaum M, Bayrak C, Zheng D, Lachman HM. 2017; CRISPR/Cas9-me-diated heterozygous knockout of the autism gene CHD8 and characterization of its transcriptional networks in cerebral organoids derived from iPS cells. Mol Autism. 8:11. DOI:
10.1186/s13229-017-0124-1. PMID:
28321286. PMCID:
PMC5357816.

27. Xu R, Brawner AT, Li S, Liu JJ, Kim H, Xue H, Pang ZP, Kim WY, Hart RP, Liu Y, Jiang P. 2019; OLIG2 drives abnormal neurodevelopmental phenotypes in human iPSC-based organoid and chimeric mouse models of down syndrome. Cell Stem Cell. 24:908–926.e8. DOI:
10.1016/j.stem.2019.04.014. PMID:
31130512. PMCID:
PMC6944064.

28. Mellios N, Feldman DA, Sheridan SD, Ip JPK, Kwok S, Amoah SK, Rosen B, Rodriguez BA, Crawford B, Swaminathan R, Chou S, Li Y, Ziats M, Ernst C, Jaenisch R, Haggarty SJ, Sur M. 2018; MeCP2-regulated miRNAs control early human neurogenesis through differential effects on ERK and AKT signaling. Mol Psychiatry. 23:1051–1065. DOI:
10.1038/mp.2017.86. PMID:
28439102. PMCID:
PMC5815944.

29. Latour YL, Yoon R, Thomas SE, Grant C, Li C, Sena-Esteves M, Allende ML, Proia RL, Tifft CJ. 2019; Human GLB1 knockout cerebral organoids: a model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis. Mol Genet Metab Rep. 21:100513. DOI:
10.1016/j.ymgmr.2019.100513. PMID:
31534909. PMCID:
PMC6744524.

30. Allende ML, Cook EK, Larman BC, Nugent A, Brady JM, Golebiowski D, Sena-Esteves M, Tifft CJ, Proia RL. 2018; Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferen-tiation. J Lipid Res. 59:550–563. DOI:
10.1194/jlr.M081323. PMID:
29358305. PMCID:
PMC5832932.

31. Gonzalez C, Armijo E, Bravo-Alegria J, Becerra-Calixto A, Mays CE, Soto C. 2018; Modeling amyloid beta and tau pathology in human cerebral organoids. Mol Psychiatry. 23:2363–2374. DOI:
10.1038/s41380-018-0229-8. PMID:
30171212. PMCID:
PMC6594704.

32. Lin YT, Seo J, Gao F, Feldman HM, Wen HL, Penney J, Cam HP, Gjoneska E, Raja WK, Cheng J, Rueda R, Kritskiy O, Abdurrob F, Peng Z, Milo B, Yu CJ, Elmsaouri S, Dey D, Ko T, Yankner BA, Tsai LH. 2018; APOE4 causes widespread molecular and cellular alterations associated with Alzheimer's disease phenotypes in human iPSC-derived brain cell types. Neuron. 98:1141–1154.e7. Erratum in: Neuron 2018;98:1294. DOI:
10.1016/j.neuron.2018.05.008. PMID:
29861287. PMCID:
PMC6023751.

33. Meyer K, Feldman HM, Lu T, Drake D, Lim ET, Ling KH, Bishop NA, Pan Y, Seo J, Lin YT, Su SC, Church GM, Tsai LH, Yankner BA. 2019; REST and neural gene network dysregulation in iPSC models of Alzheimer's disease. Cell Rep. 26:1112–1127.e9. DOI:
10.1016/j.celrep.2019.01.023. PMID:
30699343. PMCID:
PMC6386196.

34. Seo J, Kritskiy O, Watson LA, Barker SJ, Dey D, Raja WK, Lin YT, Ko T, Cho S, Penney J, Silva MC, Sheridan SD, Lucente D, Gusella JF, Dickerson BC, Haggarty SJ, Tsai LH. 2017; Inhibition of p25/Cdk5 attenuates tauopathy in mouse and iPSC models of frontotemporal dementia. J Neurosci. 37:9917–9924. DOI:
10.1523/JNEUROSCI.0621-17.2017. PMID:
28912154. PMCID:
PMC5637118.

36. Pérez-Brangulí F, Buchsbaum IY, Pozner T, Regensburger M, Fan W, Schray A, Börstler T, Mishra H, Gräf D, Kohl Z, Winkler J, Berninger B, Cappello S, Winner B. 2019; Human SPG11 cerebral organoids reveal cortical neurogenesis impairment. Hum Mol Genet. 28:961–971. DOI:
10.1093/hmg/ddy397. PMID:
30476097. PMCID:
PMC6400051.

37. Conforti P, Besusso D, Bocchi VD, Faedo A, Cesana E, Rossetti G, Ranzani V, Svendsen CN, Thompson LM, Toselli M, Biella G, Pagani M, Cattaneo E. 2018; Faulty neuronal determination and cell polarization are reverted by modulating HD early phenotypes. Proc Natl Acad Sci U S A. 115:E762–E771. Erratum in: Proc Natl Acad Sci U S A 2018;115:E2148. DOI:
10.1073/pnas.1715865115. PMID:
29311338. PMCID:
PMC5789931.

38. Chambers SM, Fasano CA, Papapetrou EP, Tomishima M, Sadelain M, Studer L. 2009; Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling. Nat Biotechnol. 27:275–280. Erratum in: Nat Biotechnol 2009;27:485. DOI:
10.1038/nbt.1529. PMID:
19252484. PMCID:
PMC2756723.

39. Kadoshima T, Sakaguchi H, Nakano T, Soen M, Ando S, Eiraku M, Sasai Y. 2013; Self-organization of axial polarity, inside-out layer pattern, and species-specific progenitor dynamics in human ES cell-derived neocortex. Proc Natl Acad Sci U S A. 110:20284–20289. Erratum in: Proc Natl Acad Sci U S A 2014;111:7498. DOI:
10.1073/pnas.1315710110. PMID:
24277810. PMCID:
PMC3864329.

40. Mariani J, Coppola G, Zhang P, Abyzov A, Provini L, Tomasini L, Amenduni M, Szekely A, Palejev D, Wilson M, Gerstein M, Grigorenko EL, Chawarska K, Pelphrey KA, Howe JR, Vaccarino FM. 2015; FOXG1-dependent dysregulation of GABA/glutamate neuron differentiation in autism spectrum disorders. Cell. 162:375–390. DOI:
10.1016/j.cell.2015.06.034. PMID:
26186191. PMCID:
PMC4519016.

41. Paşca AM, Sloan SA, Clarke LE, Tian Y, Makinson CD, Huber N, Kim CH, Park JY, O'Rourke NA, Nguyen KD, Smith SJ, Huguenard JR, Geschwind DH, Barres BA, Paşca SP. 2015; Functional cortical neurons and astrocytes from human pluripotent stem cells in 3D culture. Nat Methods. 12:671–678. DOI:
10.1038/nmeth.3415. PMID:
26005811. PMCID:
PMC4489980.

42. Qian X, Nguyen HN, Song MM, Hadiono C, Ogden SC, Hammack C, Yao B, Hamersky GR, Jacob F, Zhong C, Yoon KJ, Jeang W, Lin L, Li Y, Thakor J, Berg DA, Zhang C, Kang E, Chickering M, Nauen D, Ho CY, Wen Z, Christian KM, Shi PY, Maher BJ, Wu H, Jin P, Tang H, Song H, Ming GL. 2016; Brain-region-specific organoids using mini-bioreactors for modeling ZIKV exposure. Cell. 165:1238–1254. DOI:
10.1016/j.cell.2016.04.032. PMID:
27118425. PMCID:
PMC4900885.

43. Watanabe M, Buth JE, Vishlaghi N, de la Torre-Ubieta L, Taxidis J, Khakh BS, Coppola G, Pearson CA, Yamauchi K, Gong D, Dai X, Damoiseaux R, Aliyari R, Liebscher S, Schenke-Layland K, Caneda C, Huang EJ, Zhang Y, Cheng G, Geschwind DH, Golshani P, Sun R, Novitch BG. 2017; Self-organized cerebral organoids with human-specific features predict effective drugs to combat Zika virus infection. Cell Rep. 21:517–532. DOI:
10.1016/j.celrep.2017.09.047. PMID:
29020636. PMCID:
PMC5637483.

44. Xiang Y, Tanaka Y, Patterson B, Kang YJ, Govindaiah G, Roselaar N, Cakir B, Kim KY, Lombroso AP, Hwang SM, Zhong M, Stanley EG, Elefanty AG, Naegele JR, Lee SH, Weissman SM, Park IH. 2017; Fusion of regionally specified hPSC-derived organoids models human brain development and interneuron migration. Cell Stem Cell. 21:383–398.e7. DOI:
10.1016/j.stem.2017.07.007. PMID:
28757360. PMCID:
PMC5720381.

45. Birey F, Andersen J, Makinson CD, Islam S, Wei W, Huber N, Fan HC, Metzler KRC, Panagiotakos G, Thom N, O'Rourke NA, Steinmetz LM, Bernstein JA, Hallmayer J, Huguenard JR, Paşca SP. 2017; Assembly of functionally integrated human forebrain spheroids. Nature. 545:54–59. DOI:
10.1038/nature22330. PMID:
28445465. PMCID:
PMC5805137.

46. Qian X, Su Y, Adam CD, Deutschmann AU, Pather SR, Goldberg EM, Su K, Li S, Lu L, Jacob F, Nguyen PTT, Huh S, Hoke A, Swinford-Jackson SE, Wen Z, Gu X, Pierce RC, Wu H, Briand LA, Chen HI, Wolf JA, Song H, Ming GL. 2020; Sliced human cortical organoids for modeling distinct cortical layer formation. Cell Stem Cell. 26:766–781.e9. DOI:
10.1016/j.stem.2020.02.002. PMID:
32142682. PMCID:
PMC7366517.

47. Sloan SA, Darmanis S, Huber N, Khan TA, Birey F, Caneda C, Reimer R, Quake SR, Barres BA, Paşca SP. 2017; Human astrocyte maturation captured in 3D cerebral cortical spheroids derived from pluripotent stem cells. Neuron. 95:779–790.e6. DOI:
10.1016/j.neuron.2017.07.035. PMID:
28817799. PMCID:
PMC5890820.

48. Trujillo CA, Gao R, Negraes PD, Gu J, Buchanan J, Preissl S, Wang A, Wu W, Haddad GG, Chaim IA, Domissy A, Vandenberghe M, Devor A, Yeo GW, Voytek B, Muotri AR. 2019; Complex oscillatory waves emerging from cortical organoids model early human brain network development. Cell Stem Cell. 25:558–569.e7. DOI:
10.1016/j.stem.2019.08.002. PMID:
31474560. PMCID:
PMC6778040.

49. Bagley JA, Reumann D, Bian S, Lévi-Strauss J, Knoblich JA. 2017; Fused cerebral organoids model interactions between brain regions. Nat Methods. 14:743–751. DOI:
10.1038/nmeth.4304. PMID:
28504681. PMCID:
PMC5540177.

50. Sakaguchi H, Kadoshima T, Soen M, Narii N, Ishida Y, Ohgushi M, Takahashi J, Eiraku M, Sasai Y. 2015; Generation of functional hippocampal neurons from self-organizing human embryonic stem cell-derived dorsomedial telencephalic tissue. Nat Commun. 6:8896. DOI:
10.1038/ncomms9896. PMID:
26573335. PMCID:
PMC4660208.

51. Jacob F, Pather SR, Huang WK, Zhang F, Wong SZH, Zhou H, Cubitt B, Fan W, Chen CZ, Xu M, Pradhan M, Zhang DY, Zheng W, Bang AG, Song H, Carlos de la Torre J, Ming GL. 2020; Human pluripotent stem cell-derived neural cells and brain organoids reveal SARS-CoV-2 neurotropism predominates in choroid plexus epithelium. Cell Stem Cell. 27:937–950.e9. DOI:
10.1016/j.stem.2020.09.016. PMID:
33010822. PMCID:
PMC7505550.

52. Pomeshchik Y, Klementieva O, Gil J, Martinsson I, Hansen MG, de Vries T, Sancho-Balsells A, Russ K, Savchenko E, Collin A, Vaz AR, Bagnoli S, Nacmias B, Rampon C, Sorbi S, Brites D, Marko-Varga G, Kokaia Z, Rezeli M, Gouras GK, Roybon L. 2020; Human iPSC-derived hippocampal spheroids: an innovative tool for stratifying Alzheimer disease patient-specific cellular phenotypes and developing therapies. Stem Cell Reports. 15:256–273. Erratum in: Stem Cell Reports 2021;16:2838. DOI:
10.1016/j.stemcr.2021.10.003. PMID:
34758331. PMCID:
PMC8581187.

53. Xiang Y, Tanaka Y, Cakir B, Patterson B, Kim KY, Sun P, Kang YJ, Zhong M, Liu X, Patra P, Lee SH, Weissman SM, Park IH. 2019; hESC-derived thalamic organoids form reciprocal projections when fused with cortical organoids. Cell Stem Cell. 24:487–497.e7. DOI:
10.1016/j.stem.2018.12.015. PMID:
30799279. PMCID:
PMC6853597.

55. Huang WK, Wong SZH, Pather SR, Nguyen PTT, Zhang F, Zhang DY, Zhang Z, Lu L, Fang W, Chen L, Fernandes A, Su Y, Song H, Ming GL. 2021; Generation of hypothalamic arcuate organoids from human induced pluripotent stem cells. Cell Stem Cell. 28:1657–1670.e10. DOI:
10.1016/j.stem.2021.04.006. PMID:
33961804. PMCID:
PMC8419002.

56. Pellegrini L, Bonfio C, Chadwick J, Begum F, Skehel M, Lancaster MA. 2020; Human CNS barrier-forming organoids with cerebrospinal fluid production. Science. 369:eaaz5626. DOI:
10.1126/science.aaz5626. PMID:
32527923. PMCID:
PMC7116154.

57. Jo J, Xiao Y, Sun AX, Cukuroglu E, Tran HD, Göke J, Tan ZY, Saw TY, Tan CP, Lokman H, Lee Y, Kim D, Ko HS, Kim SO, Park JH, Cho NJ, Hyde TM, Kleinman JE, Shin JH, Weinberger DR, Tan EK, Je HS, Ng HH. 2016; Midbrain-like organoids from human pluripotent stem cells contain functional dopaminergic and neuromelanin-producing neurons. Cell Stem Cell. 19:248–257. DOI:
10.1016/j.stem.2016.07.005. PMID:
27476966. PMCID:
PMC5510242.

58. Tieng V, Stoppini L, Villy S, Fathi M, Dubois-Dauphin M, Krause KH. 2014; Engineering of midbrain organoids containing long-lived dopaminergic neurons. Stem Cells Dev. 23:1535–1547. DOI:
10.1089/scd.2013.0442. PMID:
24576173.

59. Nickels SL, Modamio J, Mendes-Pinheiro B, Monzel AS, Betsou F, Schwamborn JC. 2020; Reproducible generation of human midbrain organoids for in vitro modeling of Parkinson's disease. Stem Cell Res. 46:101870. DOI:
10.1016/j.scr.2020.101870. PMID:
32534166.

60. Muguruma K, Nishiyama A, Kawakami H, Hashimoto K, Sasai Y. 2015; Self-organization of polarized cerebellar tissue in 3D culture of human pluripotent stem cells. Cell Rep. 10:537–550. DOI:
10.1016/j.celrep.2014.12.051. PMID:
25640179.

61. Eura N, Matsui TK, Luginbühl J, Matsubayashi M, Nanaura H, Shiota T, Kinugawa K, Iguchi N, Kiriyama T, Zheng C, Kouno T, Lan YJ, Kongpracha P, Wiriyasermkul P, Sakaguchi YM, Nagata R, Komeda T, Morikawa N, Kitayoshi F, Jong M, Kobashigawa S, Nakanishi M, Hasegawa M, Saito Y, Shiromizu T, Nishimura Y, Kasai T, Takeda M, Kobayashi H, Inagaki Y, Tanaka Y, Makinodan M, Kishimoto T, Kuniyasu H, Nagamori S, Muotri AR, Shin JW, Sugie K, Mori E. 2020; Brainstem organoids from human pluripotent stem cells. Front Neurosci. 14:538. DOI:
10.3389/fnins.2020.00538. PMID:
32670003. PMCID:
PMC7332712. PMID:
df4e7359f1aa4a4388730a853f99d7c0.

63. Cakir B, Xiang Y, Tanaka Y, Kural MH, Parent M, Kang YJ, Chapeton K, Patterson B, Yuan Y, He CS, Raredon MSB, Dengelegi J, Kim KY, Sun P, Zhong M, Lee S, Patra P, Hyder F, Niklason LE, Lee SH, Yoon YS, Park IH. 2019; Engineering of human brain organoids with a functional vascular-like system. Nat Methods. 16:1169–1175. DOI:
10.1038/s41592-019-0586-5. PMID:
31591580. PMCID:
PMC6918722.

64. Shi Y, Sun L, Wang M, Liu J, Zhong S, Li R, Li P, Guo L, Fang A, Chen R, Ge WP, Wu Q, Wang X. 2020; Vascularized human cortical organoids (vOrganoids) model cortical development in vivo. PLoS Biol. 18:e3000705. DOI:
10.1371/journal.pbio.3000705. PMID:
32401820. PMCID:
PMC7250475. PMID:
66f6d290c2f84ede886185d58cd7368f.

65. Song L, Yuan X, Jones Z, Griffin K, Zhou Y, Ma T, Li Y. 2019; Assembly of human stem cell-derived cortical spheroids and vascular spheroids to model 3-D brain-like tissues. Sci Rep. 9:5977. DOI:
10.1038/s41598-019-42439-9. PMID:
30979929. PMCID:
PMC6461701.

66. Abud EM, Ramirez RN, Martinez ES, Healy LM, Nguyen CHH, Newman SA, Yeromin AV, Scarfone VM, Marsh SE, Fimbres C, Caraway CA, Fote GM, Madany AM, Agrawal A, Kayed R, Gylys KH, Cahalan MD, Cummings BJ, Antel JP, Mortazavi A, Carson MJ, Poon WW, Blurton-Jones M. 2017; iPSC-derived human microglia-like cells to study neurological diseases. Neuron. 94:278–293.e9. DOI:
10.1016/j.neuron.2017.03.042. PMID:
28426964. PMCID:
PMC5482419.

67. Cederquist GY, Asciolla JJ, Tchieu J, Walsh RM, Cornacchia D, Resh MD, Studer L. 2019; Specification of positional identity in forebrain organoids. Nat Biotechnol. 37:436–444. DOI:
10.1038/s41587-019-0085-3. PMID:
30936566. PMCID:
PMC6447454.

68. Li R, Sun L, Fang A, Li P, Wu Q, Wang X. 2017; Recapitulating cortical development with organoid culture in vitro and modeling abnormal spindle-like (ASPM related primary) microcephaly disease. Protein Cell. 8:823–833. DOI:
10.1007/s13238-017-0479-2. PMID:
29058117. PMCID:
PMC5676597.

69. Faheem M, Naseer MI, Rasool M, Chaudhary AG, Kumosani TA, Ilyas AM, Pushparaj P, Ahmed F, Algahtani HA, Al-Qahtani MH, Saleh Jamal H. 2015; Molecular genetics of human primary microcephaly: an overview. BMC Med Genomics. 8 Suppl 1:S4. DOI:
10.1186/1755-8794-8-S1-S4. PMID:
25951892. PMCID:
PMC4315316.

70. Mlakar J, Korva M, Tul N, Popović M, Poljšak-Prijatelj M, Mraz J, Kolenc M, Resman Rus K, Vesnaver Vipotnik T, Fabjan Vodušek V, Vizjak A, Pižem J, Petrovec M, Avšič Županc T. 2016; Zika virus associated with microcephaly. N Engl J Med. 374:951–958. DOI:
10.1056/NEJMoa1600651. PMID:
26862926.

71. Yoon KJ, Song G, Qian X, Pan J, Xu D, Rho HS, Kim NS, Habela C, Zheng L, Jacob F, Zhang F, Lee EM, Huang WK, Ringeling FR, Vissers C, Li C, Yuan L, Kang K, Kim S, Yeo J, Cheng Y, Liu S, Wen Z, Qin CF, Wu Q, Christian KM, Tang H, Jin P, Xu Z, Qian J, Zhu H, Song H, Ming GL. 2017; Zika-virus-encoded NS2A disrupts mammalian cortical neurogenesis by degrading adherens junction proteins. Cell Stem Cell. 21:349–358.e6. DOI:
10.1016/j.stem.2017.07.014. PMID:
28826723. PMCID:
PMC5600197.

72. Thomas CA, Tejwani L, Trujillo CA, Negraes PD, Herai RH, Mesci P, Macia A, Crow YJ, Muotri AR. 2017; Modeling of TREX1-dependent autoimmune disease using human stem cells highlights L1 accumulation as a source of neuroinflammation. Cell Stem Cell. 21:319–331.e8. DOI:
10.1016/j.stem.2017.07.009. PMID:
28803918. PMCID:
PMC5591075.

73. Reiner O. 2013; LIS1 and DCX: implications for brain development and human disease in relation to microtubules. Scientifica (Cairo). 2013:393975. DOI:
10.1155/2013/393975. PMID:
24278775. PMCID:
PMC3820303.

74. Egan MJ, Tan K, Reck-Peterson SL. 2012; Lis1 is an initiation factor for dynein-driven organelle transport. J Cell Biol. 197:971–982. DOI:
10.1083/jcb.201112101. PMID:
22711696. PMCID:
PMC3384415.

75. Karzbrun E, Kshirsagar A, Cohen SR, Hanna JH, Reiner O. 2018; Human brain organoids on a chip reveal the physics of folding. Nat Phys. 14:515–522. DOI:
10.1038/s41567-018-0046-7. PMID:
29760764. PMCID:
PMC5947782.

76. Iefremova V, Manikakis G, Krefft O, Jabali A, Weynans K, Wilkens R, Marsoner F, Brändl B, Müller FJ, Koch P, Ladewig J. 2017; An organoid-based model of cortical development identifies non-cell-autonomous defects in Wnt signaling contributing to Miller-Dieker syndrome. Cell Rep. 19:50–59. DOI:
10.1016/j.celrep.2017.03.047. PMID:
28380362.

77. Neul JL, Kaufmann WE, Glaze DG, Christodoulou J, Clarke AJ, Bahi-Buisson N, Leonard H, Bailey ME, Schanen NC, Zappella M, Renieri A, Huppke P, Percy AK. RettSearch Consortium. 2010; Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 68:944–950. DOI:
10.1002/ana.22124. PMID:
21154482. PMCID:
PMC3058521.

78. Amir RE, Van den Veyver IB, Wan M, Tran CQ, Francke U, Zoghbi HY. 1999; Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet. 23:185–188. DOI:
10.1038/13810. PMID:
10508514.

79. Lyst MJ, Bird A. 2015; Rett syndrome: a complex disorder with simple roots. Nat Rev Genet. 16:261–275. DOI:
10.1038/nrg3897. PMID:
25732612.

80. Xiang Y, Tanaka Y, Patterson B, Hwang SM, Hysolli E, Cakir B, Kim KY, Wang W, Kang YJ, Clement EM, Zhong M, Lee SH, Cho YS, Patra P, Sullivan GJ, Weissman SM, Park IH. 2020; Dysregulation of BRD4 function underlies the functional abnormalities of MeCP2 mutant neurons. Mol Cell. 79:84–98.e9. DOI:
10.1016/j.molcel.2020.05.016. PMID:
32526163. PMCID:
PMC7375197.

81. Brandon NJ, Sawa A. 2011; Linking neurodevelopmental and synaptic theories of mental illness through DISC1. Nat Rev Neurosci. 12:707–722. DOI:
10.1038/nrn3120. PMID:
22095064. PMCID:
PMC3954824.

82. Ye F, Kang E, Yu C, Qian X, Jacob F, Yu C, Mao M, Poon RYC, Kim J, Song H, Ming GL, Zhang M. 2017; DISC1 regulates neurogenesis via modulating kinetochore attachment of Ndel1/Nde1 during mitosis. Neuron. 96:1041–1054.e5. Erratum in: Neuron 2017;96:1204. DOI:
10.1016/j.neuron.2017.10.010. PMID:
29103808. PMCID:
PMC5731645.

83. Chiang CH, Su Y, Wen Z, Yoritomo N, Ross CA, Margolis RL, Song H, Ming GL. 2011; Integration-free induced pluripotent stem cells derived from schizophrenia patients with a DISC1 mutation. Mol Psychiatry. 16:358–360. DOI:
10.1038/mp.2011.13. PMID:
21339753. PMCID:
PMC4005725.

84. Burdick KE, Kamiya A, Hodgkinson CA, Lencz T, DeRosse P, Ishizuka K, Elashvili S, Arai H, Goldman D, Sawa A, Malhotra AK. 2008; Elucidating the relationship between DISC1, NDEL1 and NDE1 and the risk for schizophrenia: evidence of epistasis and competitive binding. Hum Mol Genet. 17:2462–2473. DOI:
10.1093/hmg/ddn146. PMID:
18469341. PMCID:
PMC2486442.

85. Sztainberg Y, Zoghbi HY. 2016; Lessons learned from studying syndromic autism spectrum disorders. Nat Neurosci. 19:1408–1417. DOI:
10.1038/nn.4420. PMID:
27786181.

86. Madhavan M, Nevin ZS, Shick HE, Garrison E, Clarkson-Paredes C, Karl M, Clayton BLL, Factor DC, Allan KC, Barbar L, Jain T, Douvaras P, Fossati V, Miller RH, Tesar PJ. 2018; Induction of myelinating oligodendrocytes in human cortical spheroids. Nat Methods. 15:700–706. DOI:
10.1038/s41592-018-0081-4. PMID:
30046099. PMCID:
PMC6508550.

87. Crino PB, Nathanson KL, Henske EP. 2006; The tuberous sclerosis complex. N Engl J Med. 355:1345–1356. DOI:
10.1056/NEJMra055323. PMID:
17005952.

89. Zhang B, He Y, Xu Y, Mo F, Mi T, Shen QS, Li C, Li Y, Liu J, Wu Y, Chen G, Zhu W, Qin C, Hu B, Zhou G. 2018; Differential antiviral immunity to Japanese encephalitis virus in developing cortical organoids. Cell Death Dis. 9:719. DOI:
10.1038/s41419-018-0763-y. PMID:
29915260. PMCID:
PMC6006338.

90. Fang Q, George AS, Brinkmeier ML, Mortensen AH, Gergics P, Cheung LY, Daly AZ, Ajmal A, Pérez Millán MI, Ozel AB, Kitzman JO, Mills RE, Li JZ, Camper SA. 2016; Genetics of combined pituitary hormone deficiency: roadmap into the genome era. Endocr Rev. 37:636–675. DOI:
10.1210/er.2016-1101. PMID:
27828722. PMCID:
PMC5155665.

91. Chatelain G, Fossat N, Brun G, Lamonerie T. 2006; Molecular dissection reveals decreased activity and not dominant negative effect in human OTX2 mutants. J Mol Med (Berl). 84:604–615. DOI:
10.1007/s00109-006-0048-2. PMID:
16607563.

92. Acampora D, Mazan S, Lallemand Y, Avantaggiato V, Maury M, Simeone A, Brûlet P. 1995; Forebrain and midbrain regions are deleted in Otx2-/-mutants due to a defective anterior neuroectoderm specification during gastrulation. Development. 121:3279–3290. DOI:
10.1242/dev.121.10.3279. PMID:
7588062.

93. Elliott J, Maltby EL, Reynolds B. 1993; A case of deletion 14(q22.1-->q22.3) associated with anophthalmia and pituitary abnormalities. J Med Genet. 30:251–252. DOI:
10.1136/jmg.30.3.251. PMID:
7682620. PMCID:
PMC1016311.

94. Mortensen AH, Schade V, Lamonerie T, Camper SA. 2015; Deletion of OTX2 in neural ectoderm delays anterior pituitary development. Hum Mol Genet. 24:939–953. DOI:
10.1093/hmg/ddu506. PMID:
25315894. PMCID:
PMC4834879.

95. Matsumoto R, Suga H, Aoi T, Bando H, Fukuoka H, Iguchi G, Narumi S, Hasegawa T, Muguruma K, Ogawa W, Takahashi Y. 2020; Congenital pituitary hypoplasia model demonstrates hypothalamic OTX2 regulation of pituitary progenitor cells. J Clin Invest. 130:641–654. DOI:
10.1172/JCI127378. PMID:
31845906. PMCID:
PMC6994153.

96. Ballabio C, Anderle M, Gianesello M, Lago C, Miele E, Cardano M, Aiello G, Piazza S, Caron D, Gianno F, Ciolfi A, Pedace L, Mastronuzzi A, Tartaglia M, Locatelli F, Ferretti E, Giangaspero F, Tiberi L. 2020; Modeling medulloblastoma in vivo and with human cerebellar organoids. Nat Commun. 11:583. DOI:
10.1038/s41467-019-13989-3. PMID:
31996670. PMCID:
PMC6989674. PMID:
9519a8f838f84cd89a2a82dbc13a35e4.

97. Splawski I, Timothy KW, Sharpe LM, Decher N, Kumar P, Bloise R, Napolitano C, Schwartz PJ, Joseph RM, Condouris K, Tager-Flusberg H, Priori SG, Sanguinetti MC, Keating MT. 2004; Ca(V)1.2 calcium channel dysfunction causes a multisystem disorder including arrhythmia and autism. Cell. 119:19–31. DOI:
10.1016/j.cell.2004.09.011. PMID:
15454078.

99. Angulo MA, Butler MG, Cataletto ME. 2015; Prader-Willi syndrome: a review of clinical, genetic, and endocrine findings. J Endocrinol Invest. 38:1249–1263. DOI:
10.1007/s40618-015-0312-9. PMID:
26062517. PMCID:
PMC4630255.

100. Tauber M, Hoybye C. 2021; Endocrine disorders in Prader-Willi syndrome: a model to understand and treat hypothalamic dysfunction. Lancet Diabetes Endocrinol. 9:235–246. DOI:
10.1016/S2213-8587(21)00002-4. PMID:
33647242.

101. Raja WK, Mungenast AE, Lin YT, Ko T, Abdurrob F, Seo J, Tsai LH. 2016; Self-organizing 3D human neural tissue derived from induced pluripotent stem cells recapitulate Alzheimer's disease phenotypes. PLoS One. 11:e0161969. DOI:
10.1371/journal.pone.0161969. PMID:
27622770. PMCID:
PMC5021368.

102. Yan Y, Song L, Bejoy J, Zhao J, Kanekiyo T, Bu G, Zhou Y, Li Y. 2018; Modeling neurodegenerative microenvironment using cortical organoids derived from human stem cells. Tissue Eng Part A. 24:1125–1137. DOI:
10.1089/ten.tea.2017.0423. PMID:
29361890. PMCID:
PMC6033307.

103. Kim H, Park HJ, Choi H, Chang Y, Park H, Shin J, Kim J, Lengner CJ, Lee YK, Kim J. 2019; Modeling G2019S-LRRK2 sporadic Parkinson's disease in 3D midbrain organoids. Stem Cell Reports. 12:518–531. DOI:
10.1016/j.stemcr.2019.01.020. PMID:
30799274. PMCID:
PMC6410341.

104. Smits LM, Reinhardt L, Reinhardt P, Glatza M, Monzel AS, Stanslowsky N, Rosato-Siri MD, Zanon A, Antony PM, Bellmann J, Nicklas SM, Hemmer K, Qing X, Berger E, Kalmbach N, Ehrlich M, Bolognin S, Hicks AA, Wegner F, Sterneckert JL, Schwamborn JC. 2019; Modeling Parkinson's disease in midbrain-like organoids. NPJ Parkinsons Dis. 5:5. DOI:
10.1038/s41531-019-0078-4. PMID:
30963107. PMCID:
PMC6450999.
