4.8 Article

RNA-guided endonuclease provides a therapeutic strategy to cure latent herpesviridae infection

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NATL ACAD SCIENCES
DOI: 10.1073/pnas.1410785111

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genome editing; latency; herpes virus

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  1. National Institutes of Health [U54CA151459, U01HL099995, P01CA139490, U01HL099999]

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Latent viral infection is a persistent cause of human disease. Although standard antiviral therapies can suppress active viral replication, no existing treatment can effectively eradicate latent infection and therefore a cure is lacking for many prevalent viral diseases. The prokaryotic immune system clustered regularly interspaced short palindromic repeat (CRISPR)/Cas evolved as a natural response to phage infections, and we demonstrate here that the CRISPR/Cas9 system can be adapted for antiviral treatment in human cells by specifically targeting the genomes of latent viral infections. Patient-derived cells from a Burkitt's lymphoma with latent Epstein-Barr virus infection showed dramatic proliferation arrest and a concomitant decrease in viral load after exposure to a CRISPR/Cas9 vector targeted to the viral genome.

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