Inventors:
- Oakland CA, US
Bennett Novitch - Los Angeles CA, US
Michael E. Jung - Los Angeles CA, US
Xiaoguang Liu - Los Angeles CA, US
Ranmal Samarasinghe - Los Angeles CA, US
Elena Korsakova - Los Angeles CA, US
International Classification:
C07D 277/82
A61P 25/28
C12N 5/079
G01N 33/50
Abstract:
Disclosed herein are Pifithrin compounds, methods of treating Rett Syndrome, brain fusion organoids comprising a fusion between a cerebral cortex (Cx) organoid and the ganglionic eminence (GE) organoid, one of which comprises, consists essentially of, or consists of neural cells having a loss of function mutation in the Methyl-CpG Binding Protein 2 (MECP2) gene, and methods of using the brain fusion organoid to screen for candidate compounds that treat, reduce, or inhibit the abnormal neural activities caused by MECP2-mutations.
