Gene-editing technology Crispr could be close to a human trial, with a University of Pennsylvania study receiving ethical approval from the US government's Recombinant DNA Advisory Committee.
The small study, which has been funded by ex-Facebook president Sean Parker, will augment T cells – potentially a way to treat cancer – but will be used to see whether Crispr is safe in humans, rather than being used as an actual treatment.
If successful, it could open the door for Crispr's use as a treatment for cancer and other diseases.
The trial will involve Crispr edits of T cells from 20 patients with cancer. There will be three edits: one which "inserts a gene for a protein engineered to detect cancer cells and instructs the T cells to target them", a second edit that removes a protein that might interfere with this process, and a third defensive edit. Once edited, the cells will be "infused" back into the patients.
Crispr is a genome editing tool that's more precise, efficient and flexible than existing technology. It essentially allows scientists to ‘cut and paste’ pieces of DNA sequence into a genome, Crispr transforms Cas-9 enzymes into precision engineers, matching DNA in particular cells and either cutting or repairing it.
It has already been used to edit cells in pig organs, potentially paving the way to safer transplantation of porcine organs into human bodies.
This article was originally published by WIRED UK
