Abstract

Elimination of HIV-1 requires clearance and removal of integrated proviral DNA from infected cells and tissues. Here, sequential long-acting slow-effective release antiviral therapy (LASER ART) and CRISPR-Cas9 demonstrate viral clearance in latent infectious reservoirs in HIV-1 infected humanized mice. HIV-1 subgenomic DNA fragments, spanning the long terminal repeats and the Gag gene, are excised in vivo, resulting in elimination of integrated proviral DNA; virus is not detected in blood, lymphoid tissue, bone marrow and brain by nested and digital-droplet PCR as well as RNAscope tests. No CRISPR-Cas9 mediated off-target effects are detected. Adoptive transfer of human immunocytes from dual treated, virus-free animals to uninfected humanized mice fails to produce infectious progeny virus. In contrast, HIV-1 is readily detected following sole LASER ART or CRISPR-Cas9 treatment. These data provide proof-of-concept that permanent viral elimination is possible.

Details

Title
Sequential LASER ART and CRISPR Treatments Eliminate HIV-1 in a Subset of Infected Humanized Mice
Author
Dash, Prasanta K 1 ; Kaminski, Rafal 2 ; Bella, Ramona 2 ; Su, Hang 1 ; Mathews, Saumi 1 ; Ahooyi, Taha M 2 ; Chen, Chen 2 ; Mancuso, Pietro 2 ; Sariyer, Rahsan 2 ; Ferrante, Pasquale 2 ; Donadoni, Martina 2 ; Robinson, Jake A 2 ; Sillman, Brady 1 ; Lin, Zhiyi 1 ; Hilaire, James R 1 ; Banoub, Mary 1 ; Elango, Monalisha 1 ; Nagsen Gautam 3 ; R Lee Mosley 1 ; Poluektova, Larisa Y 1 ; McMillan, JoEllyn 1 ; Bade, Aditya N 1 ; Gorantla, Santhi 1 ; Sariyer, Ilker K 2 ; Burdo, Tricia H 2 ; Won-Bin, Young 2 ; Amini, Shohreh 2 ; Gordon, Jennifer 2 ; Jacobson, Jeffrey M 2 ; Benson Edagwa 1 ; Khalili, Kamel 2 ; Gendelman, Howard E 1 

 Department of Pharmacology and Experimental Neuroscience, University of Nebraska Medical Center, Omaha, NE, USA 
 Department of Neuroscience, Lewis Katz School of Medicine at Temple University, Philadelphia, PA, USA 
 Department of Pharmaceutical Sciences, College of Pharmacy, University of Nebraska Medical Center, Omaha, NE, USA 
Pages
1-20
Publication year
2019
Publication date
Jul 2019
Publisher
Nature Publishing Group
e-ISSN
20411723
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
2251072496
Copyright
© 2019. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.