The first clinical trial using the gene editing technique CRISPR was given the go-ahead by panel from the US National Institutes of Health (NIH). The trial is aimed at determining if the technique is safe for use on human subjects. As there is much we have yet to learn about genes and their expression, it is a valid concern that modifying DNA in humans could cause serious complications – imagine fiddling around with the 1’s and 0’s that make up your computer’s operating system.

The recently discovered CRISPR technique has made editing, adding, and deleting genetic code much easier, and is the favored technique of many scientists as a result. It is especially easy to turn genes on and off using CRISPR, allowing scientists to determine specific gene function rapidly. The hope is that this technique (and others like it) will revolutionize medicine as well.

The approved trial will involve removing immune cells from cancer patients (aka t-cells or white blood cells), editing their genetic code and infusing them back into the patient. The edits will aim to provide the immune cells with a method of detecting cancerous cells, and a method of preventing cancer cells from shutting down the immune response.

The entire field of gene-editing will be effected by this trial. Serious complications from using the technique could put a damper on the entire field, dry up funding and make future approvals far more difficult. With any luck the technique will prove safe, and will give the field a new method of fighting a wide range of ailments.

 

Acknowledgments: Many thanks to John Doench his insight and comments on the story. John is the Associate Director of the Genetic Perturbation Platform at the Broad Institute of MIT and Harvard.

Managing Correspondent: Sam Dillavou

[1] http://www.nature.com/news/first-crispr-clinical-trial-gets-green-light-from-us-panel-1.20137

Photo: http://www.aati-us.com/product/fragment-analyzer/CRISPR