Article

Gene-sized DNA insertion at genomic safe harbors in human cells using a site-directed transposase

2025-09-17

Abstract excerpt

<h4>ABSTRACT</h4> Achieving precise and efficient integration of gene-sized DNA sequences into the human genome remains a major obstacle to gene therapy. Existing approaches depend on double-strand DNA breaks, which can lead to unintended genome alterations. Many monogenic diseases arise from diverse patient-specific mutations, making individualized correction impractical and underscoring the need for universal f...

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Literature Corpus work: ab7ea179-4d29-563c-9c08-342490285fcf
DOI: 10.1101/2025.09.16.676703

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