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. 2025 Jan 9;87(1):250–264. doi: 10.1097/MS9.0000000000002760

Table 1.

Basic characteristics of final selected studies

Author, year Country Study design/sample Brain tumor type iPSC technology (transduction, transfection)/viral or non-viral/iPSC source Host species (in case the study design is in vivo) Type of virus/reagent Aim of study Findings
1 Yamazoe et al., 2014 Japan Animal study/mouse/in vivo and in vitro Glioma Transduction/mouse iPSC Mouse xenograft Retrovirus Interventional/mesenchymal stem cells This study reports NSCs and MSCs as alternative therapeutic tools for malignant glioma
Another report in this study is the safer iPSC-NSCs migratory activity, which were transduced from iPSCs after neural induction
2 Ignacio Sancho-Martinez et al., 2016 USA Animal study/mice/in vivo and in vitro Glioma Transfection non-viral/hiPSCs Mice xenograft Plasmid Lipofectamine Interventional/Glioma tumor-initiating cells This study surveyed iPSCs potentiality in tumorigenesis to investigate gliomagenesis
3 Bian et al., 2018 China In vitro Glioblastoma Transfection/hiPSC Not applicable NA Modeling/Organoids This study reports distinct transcriptional profiles, and different cellular identities for MYCOE and GBM-like neoCORs
Results indicate that neoCORs remain viable and expand after renal subcapsular engrafting
4 Ogawa et al., 2018 USA Animal study/mouse/in vivo and in vitro Glioma Transfection viral/hiPSC Mouse xenograft CMV Modeling/organoids This study reports evidences that transformed cells rapidly become invasive and destroy surrounding organoid structures, overwhelming the entire organoid
In addition, this study reports that human-organoid-derived tumor cell lines or primary human-patient-derived glioblastoma cell lines can be transplanted into human cerebral organoids to establish invasive tumor-like structure
5 Huang et al., 2019 USA Animal study/mice/in vivo Medulloblastoma Transduction/hiPSC Mice xenograft Retrovirus and episomal plasmid Modeling/Medulloblastoma This study showed that NES cells derived from Gorlin syndrome patients could generate medulloblastoma because of PTCH1 mutation
6 Liu et al., 2019 UK Animal study/mice/in vivo and in vitro LGGs Transduction/hiPSC Mice xenograft Lentivirus Modeling/Low-grade gliomas This study recommends that regional chromosomal alterations may present prior to the acquisition of IDH mutations in at least some cases of LGGs
7 Plummer et al., 2019 Scotland In vitro Glioblastoma Transduction/hiPSC Not applicable Retrovirus Interventional/glioblastoma This study introduced an approach to study anticancer medication response differences
Moreover, TMZ and DOX treatments reasoned a reduction in the size of the gBS with little or no effect on the number of normal neuronal cells
8 Terada et al., 2019 Japan Animal study/mice/in vivo and in vitro Teratoid/Rhabdoid tumor Transfection viral/hiPSC Mice xenograft Sendai virus Modeling/Neural progenitor-like cells Findings in this study showed activation of the ESC-like signature in clinical specimens of AT/RTs but not medulloblastomas or glioblastomas
In addition, that c-MYC overexpression induces activation of the ESC-like signature in NPLC-derived tumors and drives tumor development with the rhabdoid phenotype
9 Ikemoto et al., 2020 Japan Animal study/mice/in vivo and in vitro Medulloblastoma/Tratoma Transfection viral/hiPSC Mice xenograft Sendai virus Modeling/Medulloblastoma This study used iPSC-derived from four Gorlin syndrome patient to clarify brain tumor cancers like basal cell carcinoma and medulloblastoma. There was not any correlation between Gorlin syndrome and Gln-iPSCs in non-medulloblastoma patients, but one of medulloblastomas demonstrated loss of PTCH1 gene
10 Koga et al., 2020 USA Animal study/mice/in vivo Glioblastoma Transfection-non-viral/hiPSC Mice xenograft Plasmid—lipofectamin Modeling/Glioblastoma This study reports mesenchymal and proneural subtype features by NF1-deleted tumors and PDGF-driven tumors, respectively, in mouse models
These cancer avatar models introduce a platform to assess human tumor development, governed by molecular subtype mutations and lineage-restricted differentiation
11 Tamura et al., 2020 Japan Animal study/mice/in vivo Glioblastoma Transduction/hiPSC Mice xenograft Lentivirus Interventional/Neural Stem/Progenitor Cells This study reports that in the presence of a prodrug GCV, hiPSC-derived NS/PCs transduced with the lentiviral vector expressing HSV-TK were able to inhibit the growth of human glioma cells, through the bystander killing effect
12 Krieger et al., 2020 Germany In vitro Glioblastoma Transfection non-viral/hiPSC Not applicable Plasmid—FuGene HD Modeling/Glioblastoma This study reports recapitulating the in vivo behavior of GBM by showing an extended network of long microtubes in tumor cells with organoids
13 Haag et al., 2021 USA Animal study/mice/in vivo DIPG Transfection non-viral/hiPSC Mice xenograft Plasmid-FuGene HD transfection reagent (Promega) Modeling/Astroglial and oligodendroglial differentiation This study showed increase in neural stem cell proliferation, and a viability reduction in H3.3-K27M DIPG cells reported in overexpression of K27M specifically in H3.3
Also increase apoptosis and proliferation primarily in NSCs occurred by H3.3-K27M
Viability decreases in iPSC, impairing pluripotency reprogramming, and effect on gene regulation with bivalent promoters reported by H3.3-K27M
14 Anastasaki et al., 2022 USA Animal study/mice/In vivo and in vitro Glioma Transfection non-viral/hiPSCs Mice xenograft NA Interventional/LGGs This study established a tractable experimental humanized platform for childhood brain tumors by elucidating the pathogenesis of and potential therapeutic opportunities
15 Baliña-Sánchez et al., 2023 Spain In vitro Brain tumors Transfection viral/hiPSC Not applicable Sendai virus Modeling/mesenchymal stromal cells This study introduces a non-invasive approach for brain tumor envisioned children’s treatment by generating personalized iMSC products
16 Linkous et al., 2019 USA In vitro Glioblastoma NA/hiPSCs Not applicable NA Modeling/Glioblastoma This study demonstrated glioblastoma invasion into organoids environment same as brain tissue and clarify its pathogenesis
17 Goranci-Buzhala et al., 2020 Germany In vitro Glioblastoma NA/hiPSCs Not applicable NA Modeling/Glioblastoma This study showed that iPSC-derived organoids as a suitable platform to investigate medulloblastoma
18 Hwang et al., 2020 France In vitro Glioblastoma Transfection viral/hiPSCs Not applicable Sendai virus Modeling/Glioblastoma This study showed c-met mutated iPSCs generated glioblastoma related genes after 90 days in comparison with other iPSCs
TMZ could be an efficient medication for c-met mutated iPSC-derived organoids
19 Cancer et al., 2019 Sweden In vitro Medulloblastoma Transduction/hiPSCs Not applicable Lentivirus Modeling/Medulloblastoma This study benefited iPSC-derived organoids to investigate role of oct4 in activation of mTOR as a metastasis inducer in tumors
20 Susanto et al., 2019 Sweden Animal study/mouse/in vitro and in vivo Medulloblastoma Transfection viral/hiPSCs Mouse xenograft Sendai virus Interventional/Medulloblastoma This study showed iPSC-derived human NES cells from a Gorlin syndrome patient which carrying a germline mutation in the sonic hedgehog receptor. PTCH1could mimic human medulloblastoma after implantation into mouse brain
21 Xue et al., 2021 USA Animal study/mice/In vitro and in vivo Medulloblastoma Transfection viral/hiPSCs Mice xenograft Sendai virus Modeling/medulloblastoma This study showed iPSC-derived medulloblastoma model utilized to evaluate cytotoxic effect of Frondoside A both in vitro and in vivo
22 Ballabio et al., 2020 Italy Animal study/mouse/in vitro and in vivo Medulloblastoma Transfection non-viral/hiPSCs Mouse xenograft Piggyback transposase Modeling/Medulloblastoma This study investigated iPSC-derived organoids to evaluate role of Otx2 and cMYC in medulloblastoma formation

AT/RTs, teratoid/rhabdoid tumors; CMV, cytomegalovirus; DIPG, diffuse intrinsic pontine glioma; DOX, doxorubicin; ESC, embryonic stem cell; GBM, glioblastoma; gBS, glioblastoma brain sphere; GCV, ganciclovir; hiPSC, human induced pluripotent stem cell; HSV-TK, herpes simplex virus thymidine kinase; iMSC, induced mesenchymal stem cell; iPSC, induced pluripotent stem cell; LGG, low-grade glioma; MSCs, mesenchymal stem cells; mTOR, mammalian target of rapamycin, MYCOE, myc oncogene-expressing cells; neoCOR, neoplastic cerebral organoid; NES, neuroepithelial stem; NPLCs, neural progenitor-like cells; NS/PCs, neural stem/progenitor cells; NSCs, neural stem cells; TMZ, temozolomide.