Article

Oligonucleotide synthesis errors are a source of untoward variation in HDR-mediated gene editing

2025-07-11

Abstract excerpt

Single-stranded oligonucleotides (ssODNs) are used as donor templates for therapeutic gene editing by CRISPR-Cas9 cleavage and homology-directed repair (HDR). Although ssODN sequence fidelity is critical to the safety and efficacy of editing, standard quality control methods cannot resolve individual nucleotide errors. By deep sequencing ssODNs from three manufacturers, and amplicons from edited hematopoietic stem...

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Literature Corpus work: 3c6da744-1a1e-57dd-a261-e5e85e1fa30c
DOI: 10.1101/2025.07.08.663753

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