A brain organoid study reveals how a genetic mutation associated with Pitt-Hopkins syndrome, a profound form of autism, disrupts neural development. Using gene-editing technology, researchers recovered the function of the TCF4 gene and effectively restored neural structure and function.
Using CRISPR-Cas9 gene editing, researchers identified actionable pathways responsible for the growth of glioblastoma stem cells. By reverse engineering brain cancer cells, multiple potential new targets for cancer treatments have been uncovered.