Modeling MECP2 Dosage in Human Cerebral Organoids

Alysson Renato Muotri, PhD | University of California San Diego

$209,000 AWARDED

Levels of MeCP2 protein are tightly controlled in the brain.  Too little leads to Rett Syndrome, whereas too much leads to another neurodevelopmental disorders. How exactly the correct levels of MeCP2 maintain the proper function of neuronal networks is an important and fundamental question. With RSRT support, Dr. Muotri and his team will genetically engineer human pluripotent stem cells to control MeCP2 levels in “mini-brains” in a dish. The team will perform a comprehensive gene expression analyses to relate molecular pathways to cellular phenotypes. The work has the potential to reveal novel therapeutic opportunities for Rett Syndrome and will also be informative for future gene therapy/protein replacement, where the amount of MeCP2 to be delivered to the brain is critical. 

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