Tuesday, November 15, 2016

CRISPR and Humans

Nature discusses the first application of CRISPR technology to humans. It was in China. They state:

On 28 October, a team led by oncologist Lu You at Sichuan University in Chengdu delivered the modified cells into a patient with aggressive lung cancer as part of a clinical trial at the West China Hospital, also in Chengdu. Earlier clinical trials using cells edited with a different technique have excited clinicians. The introduction of CRISPR, which is simpler and more efficient than other techniques, will probably accelerate the race to get gene-edited cells into the clinic across the world, says Carl June, who specializes in immunotherapy at the University of Pennsylvania in Philadelphia and led one of the earlier studies....June is the scientific adviser for a planned US trial that will use CRISPR to target three genes in participants’ cells, with the goal of treating various cancers. He expects the trial to start in early 2017. And in March 2017, a group at Peking University in Beijing hopes to start three clinical trials using CRISPR against bladder, prostate and renal-cell cancers. Those trials do not yet have approval or funding.

They note that this may become a race comparable to the space race of the 1960s. We have been following this for about three years now and the potential is significant but CRISPR Cas 9 still has the DSB problem of reassembly as well as the targeting specificity.