Crispr moves into the clinic

When Jennifer Doudna and Emmanuelle Charpentier revealed that the bacterial Crispr–Cas9 antiviral defence system could be reprogrammed to edit genomic DNA they could scarcely have imagined the impact their discovery would have. One Nobel prize and a decade later treatments based on the technique are racing towards the clinic.

That impressive speed in developing Crispr from intriguing discovery to viable therapeutic tool is perhaps not surprising, given just how big an advance it represents in our ability to manipulate genes, says Fyodor Urnov, director of technology and translation at the Innovative Genomics Institute in Berkeley, California. ‘For pretty much the first 150 years of genetics we were like astronomers. We could see the stars and galaxies, but we could never fly to them, never touch them,’ he says.

Crispr, however, turned us into astronauts. As a myriad of Crispr-based therapies for illnesses as diverse as sickle cell disease, cancer and HIV/Aids have advanced through clinical trials over the past three years they have shown that it is feasible to edit a person’s genome to cure disease. ‘This idea itself is old,’ says Urnov. ‘But we’ve never had a technology like Crispr before.’