MYCN and KAT2A form a feedforward loop to drive an oncogenic transcriptional program in neuroblastoma

Abstract The oncoprotein MYCN drives malignancy in various cancer types, including neuroblastoma (NB). However, our understanding of the mechanisms underlying its transcriptional activity and oncogenic function, as well as effective strategies to target it, remains limited. We discovered that MYCN i...

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Main Authors: Zhihui Liu, Jason J. Hong, Xiyuan Zhang, Carly M. Sayers, Wendy Fang, Man Xu, Sydney Loria, Sakereh Maskal, Haiyan Lei, Haitao Wu, Rolf Swenson, Jordan L. Meier, Jack F. Shern, Carol J. Thiele
Format: Article
Language:English
Published: Nature Publishing Group 2025-04-01
Series:Oncogenesis
Online Access:https://doi.org/10.1038/s41389-025-00557-2
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author Zhihui Liu
Jason J. Hong
Xiyuan Zhang
Carly M. Sayers
Wendy Fang
Man Xu
Sydney Loria
Sakereh Maskal
Haiyan Lei
Haitao Wu
Rolf Swenson
Jordan L. Meier
Jack F. Shern
Carol J. Thiele
author_facet Zhihui Liu
Jason J. Hong
Xiyuan Zhang
Carly M. Sayers
Wendy Fang
Man Xu
Sydney Loria
Sakereh Maskal
Haiyan Lei
Haitao Wu
Rolf Swenson
Jordan L. Meier
Jack F. Shern
Carol J. Thiele
author_sort Zhihui Liu
collection DOAJ
description Abstract The oncoprotein MYCN drives malignancy in various cancer types, including neuroblastoma (NB). However, our understanding of the mechanisms underlying its transcriptional activity and oncogenic function, as well as effective strategies to target it, remains limited. We discovered that MYCN interacts with the transcriptional coactivator KAT2A, and this interaction significantly contributes to MYCN’s activity in NB. Our genome-wide analyses indicate MYCN recruits KAT2A to bind to DNA, thereby transcriptionally regulating genes associated with ribosome biogenesis and RNA processing. Moreover, we identified that MYCN directly activates KAT2A transcription, while KAT2A acetylates MYCN, increasing MYCN protein stability. Consequently, MYCN and KAT2A establish a feedforward loop that effectively regulates global gene expression, governing the malignant NB phenotype. Treatment of NB cells with a KAT2A Proteolysis Targeting Chimera (PROTAC) degrader reduces MYCN protein levels, antagonizes MYCN-mediated gene transcription regulation and suppresses cell proliferation. This study highlights the potential of transcriptional cofactors as viable targets for developing anti-MYCN therapies.
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series Oncogenesis
spelling doaj-art-a44fb64761754bd184ac35a697b036152025-08-20T03:15:10ZengNature Publishing GroupOncogenesis2157-90242025-04-0114111310.1038/s41389-025-00557-2MYCN and KAT2A form a feedforward loop to drive an oncogenic transcriptional program in neuroblastomaZhihui Liu0Jason J. Hong1Xiyuan Zhang2Carly M. Sayers3Wendy Fang4Man Xu5Sydney Loria6Sakereh Maskal7Haiyan Lei8Haitao Wu9Rolf Swenson10Jordan L. Meier11Jack F. Shern12Carol J. Thiele13Pediatric Oncology Branch, National Cancer InstitutePediatric Oncology Branch, National Cancer InstitutePediatric Oncology Branch, National Cancer InstitutePediatric Oncology Branch, National Cancer InstitutePediatric Oncology Branch, National Cancer InstitutePediatric Oncology Branch, National Cancer InstitutePediatric Oncology Branch, National Cancer InstitutePediatric Oncology Branch, National Cancer InstitutePediatric Oncology Branch, National Cancer InstituteChemistry and Synthesis Center, National Heart, Lung, and Blood InstituteChemistry and Synthesis Center, National Heart, Lung, and Blood InstituteChemical Biology Laboratory, National Cancer InstitutePediatric Oncology Branch, National Cancer InstitutePediatric Oncology Branch, National Cancer InstituteAbstract The oncoprotein MYCN drives malignancy in various cancer types, including neuroblastoma (NB). However, our understanding of the mechanisms underlying its transcriptional activity and oncogenic function, as well as effective strategies to target it, remains limited. We discovered that MYCN interacts with the transcriptional coactivator KAT2A, and this interaction significantly contributes to MYCN’s activity in NB. Our genome-wide analyses indicate MYCN recruits KAT2A to bind to DNA, thereby transcriptionally regulating genes associated with ribosome biogenesis and RNA processing. Moreover, we identified that MYCN directly activates KAT2A transcription, while KAT2A acetylates MYCN, increasing MYCN protein stability. Consequently, MYCN and KAT2A establish a feedforward loop that effectively regulates global gene expression, governing the malignant NB phenotype. Treatment of NB cells with a KAT2A Proteolysis Targeting Chimera (PROTAC) degrader reduces MYCN protein levels, antagonizes MYCN-mediated gene transcription regulation and suppresses cell proliferation. This study highlights the potential of transcriptional cofactors as viable targets for developing anti-MYCN therapies.https://doi.org/10.1038/s41389-025-00557-2
spellingShingle Zhihui Liu
Jason J. Hong
Xiyuan Zhang
Carly M. Sayers
Wendy Fang
Man Xu
Sydney Loria
Sakereh Maskal
Haiyan Lei
Haitao Wu
Rolf Swenson
Jordan L. Meier
Jack F. Shern
Carol J. Thiele
MYCN and KAT2A form a feedforward loop to drive an oncogenic transcriptional program in neuroblastoma
Oncogenesis
title MYCN and KAT2A form a feedforward loop to drive an oncogenic transcriptional program in neuroblastoma
title_full MYCN and KAT2A form a feedforward loop to drive an oncogenic transcriptional program in neuroblastoma
title_fullStr MYCN and KAT2A form a feedforward loop to drive an oncogenic transcriptional program in neuroblastoma
title_full_unstemmed MYCN and KAT2A form a feedforward loop to drive an oncogenic transcriptional program in neuroblastoma
title_short MYCN and KAT2A form a feedforward loop to drive an oncogenic transcriptional program in neuroblastoma
title_sort mycn and kat2a form a feedforward loop to drive an oncogenic transcriptional program in neuroblastoma
url https://doi.org/10.1038/s41389-025-00557-2
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