Broad Institute
CRISPR gene editing technology is barely two years old, and it is already shaking up biotechnology and medicine. Using CRISPR biotechnologists can precisely edit genomes down to a single set of base pairs. Researchers are currently exploring just how to use this powerful new technology to treat all sorts of illnesses including sickle cell disease and cystic fibrosis.
Researchers reported in the latest issue of the Proceedings of the National Academy of Sciences how they used CRISPR to entirely extirpate HIV virus from various types of infected human cells. The PNAS editors rate the research's significance:
For more than three decades since the discovery of HIV-1, AIDS remains a major public health problem affecting greater than 35.3 million people worldwide. Current antiretroviral therapy has failed to eradicate HIV-1, partly due to the persistence of viral reservoirs. RNA-guided HIV-1 genome cleavage by the Cas9 technology has shown promising efficacy in disrupting the HIV-1 genome in latently infected cells, suppressing viral gene expression and replication, and immunizing uninfected cells against HIV-1 infection. These properties may provide a viable path toward a permanent cure for AIDS, and provide a means to vaccinate against other pathogenic viruses. Given the ease and rapidity of Cas9/guide RNA development, personalized therapies for individual patients with HIV-1 variants can be developed instantly.
Obviously, much more work needs to be done, especially on how to deliver CRISPR enabled therapies in the actual bodies of patients. Still, investigators have already applied CRISPR to cure mice of a genetic liver disease and other investigators used it to genetically modify monkeys. It seems likely that CRISPR will usher in a range of highly effective new therapies before the end of the decade.
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