NUDT21 Drives T‐Cell Acute Lymphoblastic Leukemia Through Dual Regulation of Alternative Polyadenylation and Transcriptional Activation

T-cell acute lymphoblastic leukemia (T-ALL) is an aggressive hematological malignancy with limited therapeutic options. Here, we identify the alternative polyadenylation (APA) regulator NUDT21 as a key factor in T-ALL maintenance through integrated multi-omics analyses and functional studies. NUDT21 is aberrantly upregulated in T-ALL patients, and its elevated expression is associated with poor clinical outcomes. Mechanistically, NUDT21 exerts dual oncogenic functions. As a core component of the CFIm complex, NUDT21 promotes distal poly(A) site usage of oncogenic transcripts, most prominently UBE2D3, generating long 3'UTR isoforms with enhanced mRNA stability and increased protein expression. Functionally, UBE2D3 acts as a critical downstream effector that sustains leukemia cell proliferation and survival through MYC-dependent signaling. Beyond its canonical role in APA regulation, NUDT21 also localizes to transcriptionally active promoters and interacts with lineage-specific transcription factors, including MYB, RUNX1, and GATA3, to facilitate MYC transcription. Importantly, pharmacological targeting of NUDT21 with ouabain octahydrate induces robust apoptosis in T-ALL cells by promoting NUDT21 protein degradation and concomitant suppression of UBE2D3 and MYC. Collectively, our findings establish NUDT21 as a multimodal oncogenic regulator and a promising therapeutic target in T-ALL.

Authors

Institutions

Publication Details

Journal
Advanced Science
Published
2026-04-02
DOI
https://doi.org/10.1002/advs.202520693
Primary Topic
RNA Research and Splicing
Type
article
Field-Weighted Citation Impact
0.00

Funders

Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

NUDT21 Drives T‐Cell Acute Lymphoblastic Leukemia Through Dual Regulation of Alternative Polyadenylation and Transcriptional Activation

Qi Ji, Shuiyan Wu, C.‐J. Richard Shi, Zong Zai et al.
Advanced Science
RNA Research and Splicing
article

NUDT21 Drives T‐Cell Acute Lymphoblastic Leukemia Through Dual Regulation of Alternative Polyadenylation and Transcriptional Activation

Qi Ji, Shuiyan Wu, C.‐J. Richard Shi, Zong Zai, Jinghua Yu, Yizhen Li, Haitao Zhang, Zheng Zhang, Conglian Qiu, Yijun Wu, Senlin Zhang, Jianwei Wang, Zhenjiang Bai, Zhiheng Li, Yang Yang, Jian Pan, Yanfang Tao, Shaoyan Hu
article en

Abstract

T-cell acute lymphoblastic leukemia (T-ALL) is an aggressive hematological malignancy with limited therapeutic options. Here, we identify the alternative polyadenylation (APA) regulator NUDT21 as a key factor in T-ALL maintenance through integrated multi-omics analyses and functional studies. NUDT21 is aberrantly upregulated in T-ALL patients, and its elevated expression is associated with poor clinical outcomes. Mechanistically, NUDT21 exerts dual oncogenic functions. As a core component of the CFIm complex, NUDT21 promotes distal poly(A) site usage of oncogenic transcripts, most prominently UBE2D3, generating long 3'UTR isoforms with enhanced mRNA stability and increased protein expression. Functionally, UBE2D3 acts as a critical downstream effector that sustains leukemia cell proliferation and survival through MYC-dependent signaling. Beyond its canonical role in APA regulation, NUDT21 also localizes to transcriptionally active promoters and interacts with lineage-specific transcription factors, including MYB, RUNX1, and GATA3, to facilitate MYC transcription. Importantly, pharmacological targeting of NUDT21 with ouabain octahydrate induces robust apoptosis in T-ALL cells by promoting NUDT21 protein degradation and concomitant suppression of UBE2D3 and MYC. Collectively, our findings establish NUDT21 as a multimodal oncogenic regulator and a promising therapeutic target in T-ALL.

Advanced Science
St. Jude Children's Research Hospital (US), Soochow University (CN), Children's Hospital of Suzhou University (CN)
Natural Science Foundation of Jiangsu Province, National Key Research and Development Program of China
Openalex Percentile: Top 25%
RNA Research and Splicing
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.