Tricia H Burdo, Chen Chen, Rafal Kaminski, Ilker K Sariyer, Pietro Mancuso, Martina Donadoni, Mandy D Smith, Rahsan Sariyer, Maurizio Caocci, Shuren Liao, Hong Liu, Wenwen Huo, Huaqing Zhao, John Misamore, Mark G Lewis, Vahan Simonyan, Ethan Y Xu, Thomas J Cradick, Jennifer Gordon, Kamel Khalili
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, and the Gag gene. The results presented here demonstrate that a single IV inoculation of EBT-001 at each of 3 dose levels (1...
August 17, 2023: Gene Therapy