Article

Minimization of gene editing off-target effects by tissue restriction of expression

2025-01-21

Abstract excerpt

<h4>ABSTRACT</h4> Therapeutic in vivo gene editing with highly specific nucleases has the potential to revolutionize treatment for a wide range of human diseases, including genetic disorders and latent viral infections like herpes simplex virus (HSV). However, challenges regarding specificity, efficiency, delivery, and safety must be addressed before its clinical application. A key concern is the risk of off-tar...

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Literature Corpus work: 1efef936-81bb-57c1-8e7b-82da91b02942
DOI: 10.1101/2025.01.21.634017

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