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

Wang J, Quake SR
Source: Proc Natl Acad Sci USA
Publication Date: (2014)
Issue: 111(36): 13157-62
Research Area:
Cancer Research/Cell Biology
Immunotherapy / Hematology
Cells used in publication:
Species: human
Tissue Origin:
Species: human
Tissue Origin: lung
Culture Media:
Nucleofector™ I/II/2b
a) 5x10e6 Raji or DG-75 cells, 5µg DNA Plasmids, Cell line solution V, program M-013, b)1x10e6 IMR-90 cells, 5µg of plasmid DNA, Solution V, program T-030 or X-005;
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.