MiRNAs in early brain development and pediatric cancer

The size and organization of the brain are determined by the activity of progenitor cells early in development. Key mechanisms regulating progenitor cell biology involve miRNAs. These small noncoding RNA molecules bind mRNAs with high specificity, controlling their abundance and expression. The role of miRNAs in brain development has been studied extensively, but their involvement at early stages remained unknown until recently. Here, recent findings showing the important role of miRNAs in the earliest phases of brain development are reviewed, and it is discussed how loss of specific miRNAs leads to pathological conditions, particularly adult and pediatric brain tumors. Let-7 miRNA downregulation and the initiation of embryonal tumors with multilayered rosettes (ETMR), a novel link recently discovered by the laboratory, are focused upon. Finally, it is discussed how miRNAs may be used for the diagnosis and therapeutic treatment of pediatric brain tumors, with the hope of improving the prognosis of these devastating diseases.A.P. was funded by a predoctoral fellowship from Fundación Tatiana Pérez de Guzmán el Bueno. Work in the lab was supported by grants from the European Research Council (309633) and the Spanish State Research Agency (PGC2018-102172-B-I00, as well as through the “Severo Ochoa” Programme for Centers of Excellence in R&D, ref. SEV-2017-0723).Peer reviewe