Scientific Reports · 2013 · 565 citations · 20 references
Life SciencesInfectious DiseasesMolecular BiologyGenome EditingMolecular VirologyVirologyAntiretroviral TherapyAntiviral TherapyCrispr-based EngineeringCrispr-cas SystemsTherapeutic Genome EditingVirus-induced Gene SilencingCrispr-based Genome EngineeringCrispr-cas Genome EditingCrispr-cas Target CleavageCrispr-cas TherapeuticsCrispr-cas9 Genome EditingCrispr-cas9 KnockoutDormant StagesInternal Control Genes
Even though highly active anti-retroviral therapy is able to keep HIV-1 replication under control, the virus can lie in a dormant state within the host genome, known as a latent reservoir, and poses a threat to re-emerge at any time. However, novel technologies aimed at disrupting HIV-1 provirus may be capable of eradicating viral genomes from infected individuals. In this study, we showed the potential of the CRISPR/Cas9 system to edit the HIV-1 genome and block its expression. When LTR-targeting CRISPR/Cas9 components were transfected into HIV-1 LTR expression-dormant and -inducible T cells, a significant loss of LTR-driven expression was observed after stimulation. Sequence analysis confirmed that this CRISPR/Cas9 system efficiently cleaved and mutated LTR target sites. More importantly, this system was also able to remove internal viral genes from the host cell chromosome. Our results suggest that the CRISPR/Cas9 system may be a useful tool for curing HIV-1 infection.
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Multiplex Genome Engineering Using CRISPR/Cas Systems
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RNA-Guided Human Genome Engineering via Cas9
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Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
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