Article

Genome-wide assays to characterize rAAV integration into human genomic DNA in vivo

2023-08-23

Abstract excerpt

Adeno-associated viral (AAV) vectors are used to treat genetic diseases, expressing therapeutic genes from both extrachromosomal episomes and payloads that integrate into the host genome. Assays were developed to evaluate HR-mediated on-target integration and the potential occurrence of off-target integration. While many studies have addressed elements of these processes, proper characterization requires long-read...

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Literature Corpus work: f087267d-4e43-5039-b20d-0bf6fec618e8
DOI: 10.1101/2023.08.22.554338

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