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Sci Adv ; 10(12): eadn4649, 2024 Mar 22.
Artículo en Inglés | MEDLINE | ID: mdl-38517960

RESUMEN

Genomic rearrangements are a hallmark of most childhood tumors, including medulloblastoma, one of the most common brain tumors in children, but their causes remain largely unknown. Here, we show that PiggyBac transposable element derived 5 (Pgbd5) promotes tumor development in multiple developmentally accurate mouse models of Sonic Hedgehog (SHH) medulloblastoma. Most Pgbd5-deficient mice do not develop tumors, while maintaining normal cerebellar development. Ectopic activation of SHH signaling is sufficient to enforce cerebellar granule cell progenitor-like cell states, which exhibit Pgbd5-dependent expression of distinct DNA repair and neurodevelopmental factors. Mouse medulloblastomas expressing Pgbd5 have increased numbers of somatic structural DNA rearrangements, some of which carry PGBD5-specific sequences at their breakpoints. Similar sequence breakpoints recurrently affect somatic DNA rearrangements of known tumor suppressors and oncogenes in medulloblastomas in 329 children. This identifies PGBD5 as a medulloblastoma mutator and provides a genetic mechanism for the generation of oncogenic DNA rearrangements in childhood cancer.


Asunto(s)
Neoplasias Cerebelosas , Meduloblastoma , Humanos , Niño , Animales , Ratones , Meduloblastoma/genética , Transposasas/genética , Transposasas/metabolismo , Proteínas Hedgehog/metabolismo , Factores de Transcripción/genética , Mutagénesis , Neoplasias Cerebelosas/genética
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