4.7 Article

A human forebrain organoid model of fragile X syndrome exhibits altered neurogenesis and highlights new treatment strategies

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Summary: The transcriptional silencing of the FMR1 gene in FXS leads to the loss of FMRP, which is important for regulating mRNA translation and protein synthesis. Abnormal protein synthesis in FXS is related to altered cell fate decisions favoring proliferation over neurogenesis during early development. Pharmacologic inhibition of elevated PI3K signaling can correct excess protein synthesis and cell proliferation in a subset of patient neural cells.

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