Pediatric high-grade gliomas are particularly aggressive brain tumors with a very poor prognosis. In these tumors, which are often mutated for the H3.3 variant of histone H3, a defect in a PNKP-dependent DNA repair mechanism has been identified. The resulting genomic instability may promote the transformation of cancer cells. The inventors have shown that inhibition of PNKP gene expression specifically prevents the proliferation of glioma tumor cells carrying the H3 oncohistone mutations. These promising results pave the way for targeting PNKP as a novel therapeutic strategy.
WO2023/198932
Pediatric high-grade gliomas PNKP - Histone - Synthetic lethality - H3.3 oncohistone mutations - DNA repair
Erganeo is at your disposal.
