Potent and Targeted Activation of Latent HIV-1 Using the CRISPR/dCas9 Activator Complex

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Saayman, Sheena M
Lazar, Daniel C
Scott, Tristan A
Hart, Jonathan R
Takahashi, Mayumi
Burnett, John C
Planelles, Vicente
Morris, Kevin V
Weinberg, Marc S
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2016
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Abstract

HIV-1 provirus integration results in a persistent latently infected reservoir that is recalcitrant to combined antiretroviral therapy (cART) with lifelong treatment being the only option. The "shock and kill" strategy aims to eradicate latent HIV by reactivating proviral gene expression in the context of cART treatment. Gene-specific transcriptional activation can be achieved using the RNA-guided CRISPR-Cas9 system comprising single guide RNAs (sgRNAs) with a nuclease-deficient Cas9 mutant (dCas9) fused to the VP64 transactivation domain (dCas9-VP64). We engineered this system to target 23 sites within the long terminal repeat promoter of HIV-1 and identified a "hotspot" for activation within the viral enhancer sequence. Activating sgRNAs transcriptionally modulated the latent proviral genome across multiple different in vitro latency cell models including T cells comprising a clonally integrated mCherry-IRES-Tat (LChIT) latency system. We detected consistent and effective activation of latent virus mediated by activator sgRNAs, whereas latency reversal agents produced variable activation responses. Transcriptomic analysis revealed dCas9-VP64/sgRNAs to be highly specific, while the well-characterized chemical activator TNFα induced widespread gene dysregulation. CRISPR-mediated gene activation represents a novel system which provides enhanced efficiency and specificity in a targeted latency reactivation strategy and represents a promising approach to a "functional cure" of HIV/AIDS.

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Molecular Theory

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24

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3

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© 2016 The Author(s). This work is licensed under a Creative Commons Attribution-NonCommercial-ShareAlike 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-sa/4.0/

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Biological sciences

Biomedical and clinical sciences

Genetics

Clinical sciences

Medical biotechnology

Science & Technology

Life Sciences & Biomedicine

Biotechnology & Applied Microbiology

Genetics & Heredity

Medicine, Research & Experimental

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Saayman, SM; Lazar, DC; Scott, TA; Hart, JR; Takahashi, M; Burnett, JC; Planelles, V; Morris, KV; Weinberg, MS, Potent and Targeted Activation of Latent HIV-1 Using the CRISPR/dCas9 Activator Complex, Molecular Theory, 2016, 24 (3), pp. 488-498

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