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NKX2-1 regulates cell survival, maturation, and DNA-damage responses as a cofactor of RUNX1 in T-cell acute lymphoblastic leukemia.

Title: NKX2-1 regulates cell survival, maturation, and DNA-damage responses as a cofactor of RUNX1 in T-cell acute lymphoblastic leukemia.
Authors: Van Aerschot, Linde; Demeyer, Sofie; Timcheva, Kalina; Heylen, Elien; Verstraete, Paulien; De Groote, Dylan; Caruso, Marino; Lauwereins, Lukas; Bacquelaine Veloso, Alexandra; Kampen, Kim R; Pepe, Daniele; Boeckx, Nancy; Royaert, Jonathan; Verbeeck, Jelle; Segers, Heidi; Cools, Jan; De Keersmaecker, Kim; Granados, David Cabrerizo
Source: Haematologica (2026-01-08)
Publisher Information: Ferrata Storti Foundation (Haematologica)
Publication Year: 2026
Collection: University of Liège: ORBi (Open Repository and Bibliography)
Subject Terms: Human health sciences; Hematology; Oncology; Sciences de la santé humaine; Hématologie; Oncologie
Description: peer reviewed ; T-cell acute lymphoblastic leukemia (T-ALL) is characterized by ectopic expression of transcription factors, including NKX2-1, which is overexpressed in 5% of patients. NKX2-1 is associated with a cortical immunophenotype and drives metabolic addiction to the serine/glycine synthesis pathway in T-ALL. However, a complete picture of the role of NKX2-1 in T-ALL pathogenesis is missing. We characterized a CRISPR-Cas9 NKX2-1 knockout model of RPMI-8402, the only known NKX2-1-expressing T-ALL cell line, and validated obtained results in patient samples. NKX2-1 knockout caused a less mature immunophenotype and promoted cell cycle progression, in line with direct transcriptional repression of CDK6 by NKX2-1 that we observed. Furthermore, NKX2-1 protected T-ALL cells from apoptosis and DNA damage. The NKX2-1 protein directly bound DNA repair factors, such as RPA1 and RPA2, and presence of NKX2-1 resulted in differential expression of gene sets related to DNA damage repair in RPMI-8402 cells and patient samples. Furthermore, NKX2-1 positive cells showed less induction of DNA damage and apoptosis upon treatment with etoposide, a DNA damaging chemotherapy agent that is clinically used to treat T-ALL. Mechanistically, our data supported that RUNX1 is an important co-factor for NKX2-1 transcriptional regulation in T-ALL cells, and that NKX2-1 modulated the composition of RUNX1 protein complexes. Notably, NKX2-1 expressing cells showed higher sensitivity towards RUNX1 inhibition, suggesting a cooperative role in regulating T-ALL cell survival. This work reveals a critical role of NKX2-1 in enhancing T-ALL cell survival through DNA damage protection, and identifies RUNX1 as an important cofactor in T-ALL pathogenesis.
Document Type: article in journal/newspaper
Language: English
ISSN: 0390-6078; 1592-8721
Relation: https://haematologica.org/article/download/13077/79334; urn:issn:0390-6078; urn:issn:1592-8721; https://orbi.uliege.be/handle/2268/339631; info:hdl:2268/339631; info:pmid:41504233
DOI: 10.3324/haematol.2025.287966
Availability: https://orbi.uliege.be/handle/2268/339631; https://orbi.uliege.be/bitstream/2268/339631/1/NKX2-1%20regulates%20cell%20survival%2c%20maturation%2c%20and%20DNA-damage%20responses%20as%20a%20cofactor%20of%20RUNX1%20in%20T-cell%20acute%20lymphoblastic%20leukemia.pdf; https://doi.org/10.3324/haematol.2025.287966
Rights: open access ; http://purl.org/coar/access_right/c_abf2 ; info:eu-repo/semantics/openAccess
Accession Number: edsbas.44168E94
Database: BASE