Article

Precision and efficacy of RNA-guided DNA integration in high-expressing muscle loci

2024-03-19

Abstract excerpt

Gene replacement therapies in genetic medicine primarily rely on adeno-associated viral (AAV) vectors for transgene expression. However, episomal expression can decline over time due to epigenetic silencing. CRISPR-based integration methods offer promise for long-term transgene insertion. While the development of transgene integration methods has made substantial progress, identifying optimal insertion loci remain...

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Literature Corpus work: 7f5b6570-7d54-5391-83ea-c9c6152979e5
DOI: 10.1101/2024.03.18.582796

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