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DNA polymerase theta-mediated DNA repair is a functional dependency and therapeutic vulnerability in DNMT3A deficient leukemia cells

Le, B. V.; Vekariya, U.; Toma, M. M.; Nieborowska-Skorska, M.; Caron, M.-C.; Gozdecka, M.; Haydar, Z.; Walsh, M.; Ghosh, J.; Vaughan-Williams, E.; Podszywalow-Bartnicka, P.; Kukuyan, A.-M.; Ziolkowska, S.; Hadzijusufovic, E.; Chandramouly, G.; Piwocka, K.; Pomerantz, R.; Vassiliou, G. S.; Huntly, B. J.; Valent, P.; Bellacosa, A.; Masson, J.-Y.; Gupta, G. P.; Challen, G. A.; Skorski, T.

2024-09-19 cancer biology
10.1101/2024.09.15.613155 bioRxiv
Show abstract

Myeloid malignancies carrying somatic DNMT3A mutations (DNMT3Amut) may be refractory to standard therapy. DNMT3Amut leukemia cells accumulate toxic DNA double strand breaks (DSBs) and stalled replication forks, rendering them dependent on DNA damage response (DDR). We report here that DNA polymerase theta (Pol{theta}), a key element in DSB repair by end-joining (TMEJ) and in fork restarting, is essential for survival and proliferation of DNMT3Amut leukemia cells. Pol{theta} is overexpressed in DNMT3Amut leukemia cells due to abrogation of PARP1 PARylation-dependent UBE2O E3 ligase-mediated ubiquitination and proteasomal degradation of Pol{theta}. In addition, PARP1-mediated recruitment of the SMARCAD1-MSH2/MSH3 repressive complex to DSBs was diminished in DNMT3Amut leukemia cells which facilitated loading of Pol{theta} on DNA damage and promoting TMEJ and replication fork restart. Pol{theta} inhibitors enhanced the anti-leukemic effects of standard drugs such as FLT3 kinase inhibitor quizartinib, cytarabine +/- doxorubicin, and etoposide in vitro and in mice with DNMT3Amut leukemia. Altogether, Pol{theta} is an attractive target in DNMT3Amut hematological malignancies.

Published in Cell Reports Medicine · training set

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