I was recording a new BioChat (you should subscribe) recently with a professor at Harvard. We discussed gene therapy in passing for the disease he was studying, and one of the things that he brought up was the need to ensure that whatever therapy is designed has to be safe and effective. This of course is the promise and also the challenge of CRISPR-based research, in which the model is known but the targeting efficiency isn't where it needs to be in order to be of practical use in therapeutics. This is a big reason why the biomedical research world was abuzz with the recent announcement (and preprint) of a major discovery by scientists at the Broad Institute, where they characterized the mechanisms and the potential utility of a system similar to CRISPR in eukaryotes. 

Towards the end of my doctoral research, I first heard the rumblings of an acronym termed “CRISPR” that was starting to gather momentum. By the time I earned my doctorate, the applications that were discussed in both theory and in practice accelerated to the point that, while I didn’t fully understand the mechanism of the factors involved, I was certain that the discovery and re-engineering of this prokaryotic phenomenon would eventually be recognized with a Nobel Prize. Less than a decade after their first publications on the topic, ^{1, 2} Emmanuelle Charpentier and Jennifer Doudna were awarded the Nobel Prize in Chemistry “for the development of a method for genome editing,” which sounds a lot less important than it actually is!