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Joint single-cell profiling of Cas9 edits and transcriptomes reveals on- and off-target effects on gene expression (RNA-seq)

GSE313958 Homo sapiens Expression profiling by high throughput sequencing 6 samples Submitted 2025/12/17 Platform GPL22790
Summary
A longstanding barrier in genome engineering with CRISPR-Cas9 has been the inability to characterize editing outcomes at single-cell resolution. We present "Superb-seq", a new scRNA-seq method that measures on-target and off-target genome edits and associated transcriptomes. In contrast to Perturb-seq that captures single-cell guide information, Superb-seq directly captures single-cell edit profiles by T7 in situ transcription. We demonstrate and validate this method through two Superb-seq experiments in 30,000 cells of three cell types, amplicon sequencing (rhAmpSeq), and bulk RNA-seq. Superb-seq uses off-the-shelf kits, standard laboratory equipment, and requires no virus, which will improve CRISPR screens in diverse tissues and functional characterization of CRISPR therapeutics.
Published in
Joint single-cell profiling of Cas9 edits and transcriptomes reveals widespread off-target events and effects on gene expression
Lorenzini MH, Balderson B, Sajeev K et al. · bioRxiv : the preprint server for biology 2025 · PMID 40909645 · doi:10.1101/2025.02.07.636966
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Also filed as BioProject PRJNA1390207 and SRA study SRP656069. Searching any of these in the dataset finder brings you back here.

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