Arguably, nowhere has there been a greater advance in our understanding of biological mechanisms and potential translational targets during the next-generation sequencing era than in paediatric brain tumours. The so-called omics revolution, enabled by high-throughput sequencing, has empowered large consortia and independent groups alike to make major genetic discoveries, from dominant-negative histone mutations and hijacking of distal enhancer elements, to new oncogenic gene fusions and aberrantly active gene expression. Epigenetic deregulation has also been revealed as a common theme across several tumour subtypes. This Review focuses on key findings that have been transforming the landscape of paediatric neuro-oncology research and how these results are opening new avenues towards potential therapeutic translation.
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