title: Next-generation (epi)genetic drivers of childhood brain tumours and the outlook for targeted therapies.
abstract: 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.
authors.affiliation: Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany. Electronic address: paul.northcott@stjude.org.
authors.forename: Paul A
authors.initials: PA
authors.lastname: Northcott
authors.affiliation: Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany; Department of Pediatric Oncology, Hematology & Immunology, Heidelberg University Hospital, Heidelberg Germany.
authors.forename: Stefan M
authors.initials: SM
authors.lastname: Pfister
authors.affiliation: Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), Heidelberg, Germany.
authors.forename: David T W
authors.initials: DT
authors.lastname: Jones
mesh_terms: D001932:Brain Neoplasms / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D002648:Child; D019175:DNA Methylation / Q000235:genetics; D015972:Gene Expression Regulation, Neoplastic; D050939:Gene Fusion; D059014:High-Throughput Nucleotide Sequencing; D006801:Humans; D008527:Medulloblastoma / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D058990:Molecular Targeted Therapy; D009363:Neoplasm Proteins / Q000096:biosynthesis
chemical_list: D009363:Neoplasm Proteins