Article

Curative levels of endogenous gene replacement achieved in non-human primate liver using programmable genomic integration

2024-10-13

Abstract excerpt

The ability to efficiently place a large piece of DNA in a specific genomic location has been a goal for the gene therapy field since its inception; however, despite significant advances in gene editing technology, this had yet to be achieved. Here we describe two methods of programmable genomic integration (PGI) that overcome some of the limitations of current approaches. Using a combination of clinically validat...

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Literature Corpus work: 2b636f08-d705-5d44-adfb-65a41f52e0a1
DOI: 10.1101/2024.10.12.617700

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