First CRISPR Human Clinical Trial Gets a Green Light from the U.S., by Sara Reardon.
CRISPR, the genome-editing technology that has taken biomedical science by storm, is finally nearing human trials.
“Cell therapies [for cancer] are so promising but the majority of people who get these therapies have a disease that relapses,” says study leader Edward Stadtmauer, a physician at the University of Pennsylvania in Philadelphia. Gene editing could improve such treatments and eliminate some of their vulnerabilities to cancer and the body’s immune system, he says.
This first trial is small and designed to test whether CRISPR is safe for use in people, rather than whether it cures cancer or not.
The researchers will remove T cells from 18 patients with melanoma, sarcoma or myeloma, and perform three CRISPR edits on them. One edit will insert a gene for a protein engineered to detect cancer cells and instruct the T cells to target them, and a second edit removes a natural T-cell protein that could interfere with this process. The third is defensive: it will remove the gene for a protein that identifies the T cells as immune cells and prevent the cancer cells from disabling them. The researchers will then infuse the edited cells back into the patient.
CRISPR: gene editing is just the beginning, by Heidi Ledford.
What CRISPR offers, and biologists desire, is specificity: the ability to target and study particular DNA sequences in the vast expanse of a genome. And editing DNA is just one trick that it can be used for. Scientists are hacking the tools so that they can send proteins to precise DNA targets to toggle genes on or off, and even engineer entire biological circuits — with the long-term goal of understanding cellular systems and disease.