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Article

Rational engineering of minimally immunogenic nucleases for gene therapy

2 Jan 2025

Abstract excerpt

Genome editing using CRISPR-Cas systems is a promising avenue for the treatment of genetic diseases. However, cellular and humoral immunogenicity of genome editing tools, which originate from bacteria, complicates their clinical use. Here we report reduced immunogenicity (Red)(i)-variants of two clinically relevant nucleases, SaCas9 and AsCas12a. Through MHC-associated peptide proteomics (MAPPs) analysis, we...

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PMID: 39747875
DOI: 10.1038/s41467-024-55522-1

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