Disease Modeling with iPSCs to Accelerate Drug Discovery

Rett Syndrome (RTT) is a rare autosomal dominant neurodevelopmental disorder arising from spontaneous mutations in the MECP2 gene on the X chromosome. Individuals with RTT experience severe neurological complications including progressive loss of motor skill, speech, and behavioral functions. To identify potential therapeutics for treating Rett Syndrome, we generated and screened iPSC-derived microBrain 3D spheroids from healthy control and patient donors to identify compounds and target pathways that rescued the patient derived microBrain 3D-RTT phenotype.

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