Multiplexed <i>in vivo</i> base editing identifies functional gene-variant-context interactions.

ABSTRACT: Human genome sequencing efforts in healthy and diseased individuals continue to identify a broad spectrum of genetic variants associated with predisposition, progression, and therapeutic outcomes for diseases like cancer^1-6. Insights derived from these studies have significant potential to guide clinical diagnoses and treatment decisions; however, the relative importance and functional impact of most genetic variants remain poorly understood. Precision genome editing technologies like base and prime editing can be used to systematically engineer and interrogate diverse types of endogenous genetic variants in their native context^7-9. We and others have recently developed and applied scalable sensor-based screening approaches to engineer and measure the phenotypes produ

SUBMITTER: Acosta J 

PROVIDER: S-EPMC11888363 | biostudies-literature | 2025 Feb

REPOSITORIES: biostudies-literature