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Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates

16 Aug 2023

Abstract excerpt

Abstract In this study, we demonstrate the safety and utility of CRISPR-Cas9 gene editing technology for in vivo editing of proviral DNA in ART-treated, virally controlled simian immunodeficiency virus (SIV) infected rhesus macaques, an established model for HIV infection. EBT-001 is an AAV9-based vector delivering SaCas9 and dual guide RNAs designed to target multiple regions of the SIV genome: the viral LTRs,...

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PMID: 37587230
DOI: 10.1038/s41434-023-00410-4

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