Article

AAV-delivered CRISPR-Cas9 elicits persistent retinal immune responses compared with transient responses to RNP

2025-12-14

Abstract excerpt

<h4>ABSTRACT</h4> CRISPR-Cas9 is a powerful gene-editing tool with great potential for treating genetic diseases, including inherited retinal disorders. However, its bacterial origin can induce immune responses that may eliminate transduced cells, threatening editing efficiency. A deeper understanding of CRISPR-Cas9 immunogenicity is therefore needed. Previous studies have shown that systemic delivery via Cas9 in...

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Literature Corpus work: d8c7828d-0dd2-55ce-852a-6396b40b4e72
DOI: 10.64898/2025.12.11.693665

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