Precise measurement of CRISPR genome editing outcomes through single-cell DNA sequencing.

ABSTRACT: Gene therapy for clinical applications necessitates a comprehensive, accurate, and precise measurement of gene-edited drug products. State-of-the-art pipelines for evaluating editing outcomes rely primarily on bulk sequencing approaches, which are limited to population-level assessment. Here, we leveraged Tapestri, a single-cell sequencing technology for an in-depth analysis of editing outcomes. Using this platform, we characterized the genotype of triple-edited cells simultaneously at more than 100 loci, including editing zygosity, structural variations, and cell clonality. Our findings revealed a unique editing pattern in nearly every edited cell, highlighting the importance of single-cell resolution measurement to ensure the highest safety standards.

SUBMITTER: Kalter N 

PROVIDER: S-EPMC11987616 | biostudies-literature | 2025 Jun

REPOSITORIES: biostudies-literature