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@ARTICLE{Wang:298341,
author = {C. Wang$^*$ and N. Stöffler$^*$ and H. Liu$^*$},
title = {{L}aboratory-{E}ngineered {G}lioblastoma {O}rganoid
{C}ulture and {D}rug {S}creening.},
journal = {JoVE journal},
volume = {215},
issn = {1940-087X},
address = {Cambridge, MA},
publisher = {JoVE},
reportid = {DKFZ-2025-00245},
pages = {e67593},
year = {2025},
note = {#EA:A240#LA:A240#},
abstract = {Glioblastoma (GBM) is described as a group of highly
malignant primary brain tumors and stands as one of the most
lethal malignancies. The genetic and cellular
characteristics of GBM have been a focal point of ongoing
research, revealing that it is a group of heterogeneous
diseases with variations in RNA expression, DNA methylation,
or cellular composition. Despite the wealth of molecular
data available, the lack of transferable pre-clinic models
has limited the application of this information to disease
classification rather than treatment stratification.
Transferring the patients' genetic information into clinical
benefits and bridging the gap between detailed descriptions
of GBM, genotype-phenotype associations, and treatment
advancements remain significant challenges. In this context,
we present an advanced human GBM organoid model, the
Laboratory Engineered Glioblastoma Organoid (LEGO), and
illustrate its use in studying the genotype-phenotype
dependencies and screening potential drugs for GBM.
Utilizing this model, we have identified lipid metabolism
dysregulation as a critical milestone in GBM progression and
discovered that the microsomal triglyceride transfer protein
inhibitor Lomitapide shows promise as a potential treatment
for GBM.},
keywords = {Glioblastoma: genetics / Glioblastoma: pathology / Humans /
Organoids: metabolism / Organoids: drug effects / Brain
Neoplasms: genetics / Brain Neoplasms: pathology / Drug
Screening Assays, Antitumor: methods / Drug Evaluation,
Preclinical: methods},
cin = {A240},
ddc = {570},
cid = {I:(DE-He78)A240-20160331},
pnm = {311 - Zellbiologie und Tumorbiologie (POF4-311)},
pid = {G:(DE-HGF)POF4-311},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:39868688},
doi = {10.3791/67593},
url = {https://inrepo02.dkfz.de/record/298341},
}