Comprehensive assessment of on- and off-target mutagenesis via lipid nanoparticle delivery of CRISPR-Cas9 genome editing

Molecular Therapy — Nucleic Acids 2026 Open access

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Authors: Youichi Naoe, Naoko Fujimoto, Yukimasa Makita, Dongyang Li, Naoto Inukai, Akitsu Hotta
DOI: 10.1016/j.omtn.2026.102958
Cited by: 1
References: 58

Abstract

WGS clusters yielded 11 high-confidence off-target candidates, predominantly associated with one gRNA. While sensitivity was maximized, reproducibility across conditions remained limited, and many candidate sites overlapped repetitive or low-mappability regions. Our multilayered framework demonstrates both the utility and current limitations of off-target risk assessment, providing a practical foundation for the safety evaluation of genome editing therapies.

Fields of Study & Subjects

CRISPR and Genetic Engineering RNA Interference and Gene Delivery Advanced biosensing and bioanalysis techniques

Publication details

Journal / source
Molecular Therapy — Nucleic Acids
Publisher
Elsevier BV
Volume
37
Issue
2
Pages
102958–102958
Publication date
20 May 2026
Language
EN