Targeting oncogenic fusion-driven NUT carcinoma with CRISPR-Cas9 genome editing.

ABSTRACT: NUT carcinoma (NC) is a highly aggressive malignancy characterized by an oncogenic fusion gene incorporating the NUTM1 gene. To date, no established treatment options exist. CRISPR-Cas9 technology allows precise genomic targeting, thereby presenting a promising therapeutic strategy for cancers with well-defined genomic alterations such as oncogenic fusion genes. In this study, we investigated the effects of CRISPR-Cas9-mediated disruption of the BRD4::NUTM1 fusion gene in NC cell lines using multiple single guide RNAs (sgRNAs) to target different sites of both fusion partner genes. Our experiments identified promising sgRNA candidates that were shown to successfully disrupt the BRD4::NUTM1 fusion gene at the DNA level, thus leading to an efficient knockout of the aberr

SUBMITTER: Carle MF 

PROVIDER: S-EPMC12581649 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature