Modeling with interference

While much of the focus on CRISPR-based gene editing has been on its potential to change the way genetic disorders are treated, the biggest and most immediate impact of the rapidly emerging technology so far has been on advancing preclinical research through better disease models. By coupling CRISPR interference (CRISPRi) - a variant of CRISPR-Cas9 - with induced pluripotent stem (iPS) cells, a team from the University of California San Francisco has designed a better way to model diseases and screen for drug targets.

Since the discovery that CRISPR-Cas9 was capable of editing human cells less than four years ago, rights to the technology have been tied up in a legal battle over inventorship that could ultimately determine which inventors, and affiliated companies, are allowed to commercialize therapeutics based on the modality. But while ownership over therapeutic commercialization rights is under dispute, the IP controversy hasn't restricted research use of the tool. ...