BioCentury
ARTICLE | Cover Story

CRISPR in the liver

April 24, 2014 7:00 AM UTC

Despite the fact that the therapeutic utility of CRISPR-based approaches has yet to be demonstrated, venture dollars keep flowing into new companies developing the platform. But proof of concept may come faster than expected as new findings show that a CRISPR-based compound can correct a mutation in adult mice with genetic liver disease.1

Last year, multiple independent teams adapted a newly identified bacterial defense system to create a platform for site-directed genome editing.2-7 The platform relies on a synthetic single guide RNA (sgRNA) to target the activity of the CRISPR (clustered, regularly interspaced short palindromic repeats)-associated bacterial endonuclease Cas9-the system is referred to in short as CRISPR-Cas9...