Article

Frequent, context-dependent effects of human genetic variation on Cas9 activity revealed by population-scale GUIDE-seq-2 and deep combinatorial CHANCE-seq profiling

2025-02-12

Abstract excerpt

Genome editing enzymes can introduce targeted changes to DNA in living cells 1–4 , transforming biological research and enabling the first approved gene editing therapy for sickle cell disease 5 . However, their activity can be altered by genetic variation at on- or off-target sites 6–8 , potentially impacting both their precision and therapeutic safety. Due to a lack of scalable methods to measure genome-wide...

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Literature Corpus work: d1188b68-c2b9-529c-a9c7-be6954e67ed3
DOI: 10.1101/2025.02.10.637517

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