GSK3B Inhibition Improves Myelination in APOE4 Brain Organoids

Researchers linked APOE4-associated lipid accumulation in human oligodendrocytes to altered Wnt/GSK3B signaling. A GSK3B inhibitor reduced lipid droplets in cell cultures and increased myelination in brain organoids; the findings remain preclinical.

MIT’s Stata Center in Cambridge, Massachusetts.
MIT’s Stata Center, photographed in 2022. (Corey Leopold, CC BY 2.0)

Tracing a Lipid Phenotype to Wnt Signaling

An MIT-led team used a genome-wide CRISPR screen in oligodendrocytes derived from human induced pluripotent stem cells (iPSCs) carrying APOE4, an Alzheimer’s disease risk variant.

According to MIT’s report, the researchers also compared chromatin accessibility in cultured cells and postmortem human brain samples. Together, the analyses implicated reduced Wnt signaling and overactivity of the kinase glycogen synthase kinase-3 beta (GSK3B) in lipid-droplet accumulation in these myelin-producing cells.

Testing the Target in Human Models

In APOE4 oligodendrocyte cultures, the GSK3B inhibitor CHIR99021 reduced lipid droplets. In three-dimensional organoid cultures combining APOE4 oligodendrocytes with neurons, GSK3B inhibition increased myelination and axon growth, according to MIT’s report.

Separate Evidence from Mice

The study also tested GSK3B inhibition in APOE4;PS19 tau-model mice. In the hippocampus, treatment reduced lipid droplets and increased myelination compared with untreated controls.

Sources

  1. Genome-wide CRISPR screen reveals that Wnt signaling regulates lipid accumulation in APOE4 oligodendrocytes. Cell Stem Cell. 2026-10-15. DOI: 10.1016/j.stem.2026.09.012.
  2. How the biggest Alzheimer’s risk gene disrupts the process of insulating neurons. The Picower Institute at MIT. 2026-10-15.