Screening Splice-Switching Antisense Oligonucleotides in Pancreas-Cancer Organoids.

ABSTRACT: Aberrant alternative splicing is emerging as a cancer hallmark and a potential therapeutic target. It is the result of dysregulated or mutated splicing factors, or genetic alterations in splicing-regulatory cis-elements. Targeting individual altered splicing events associated with cancer-cell dependencies is a potential therapeutic strategy, but several technical limitations need to be addressed. Patient-derived organoids are a promising platform to recapitulate key aspects of disease states, and to facilitate drug development for precision medicine. Here, we report an efficient antisense-oligonucleotide (ASO) lipofection method to systematically evaluate and screen individual splicing events as therapeutic targets in pancreatic ductal adenocarcinoma organoids. This optimized delive

SUBMITTER: Wan L 

PROVIDER: S-EPMC11387002 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature