Researchers in China have, for the first time, injected genes edited by CRISPR technology into a human test subject, the journal Nature reports. They hope the modified genes will provide a major breakthrough in the treatment of certain types of cancer.
The team of researchers led by oncologist Lu You of Sichuan University in Chengdu injected the patient with the edited cells on October 28. The patient suffers from aggressive lung cancer and is participating in a clinical trial at Chengdu’s West China Hospital, according to Nature.
“CRISPR” is an acronym for “clustered regularly interspaced short palindromic repeats.”
Scientists began experimenting with gene technology 40 years ago, Bernie Hobbs explains in an April article from the Australian Broadcasting Commission (ABC). The field has since produced a “wealth of genetic advances,” including the mapping of the human genome in 2001.
“That’s nothing compared to the genetic revolution that we’re at the beginning of right now, thanks to a technique adapted from bacteria called CRISPR,” says Hobbs. According to Hobbs, “researchers learn what genes do by switching them on or off, or cutting them out of the DNA in a cell entirely.” This type of manipulation is referred to as ‘editing’.” CRISPR was first introduced in 2012 and has already “completely transformed the process” researchers use to edit DNA.
“It’s not the first method devised for this kind of genome editing, but CRISPR is a lot cheaper, faster, and more accurate than any of the alternatives,” Hobbs explains. Researchers learned of CRISPR from studying bacteria, which use CRISPR RNA and a nuclease called Cas9 to combat viruses.
Lu’s trials at West China Hospital follow previous clinical trials involving genes edited by different techniques. Those studies ‘excited clinicians’, according to Nature. The enhanced potential of CRISPR makes this study even more thrilling.
“I think this is going to trigger ‘Sputnik 2.0’, a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product,” Carl June, a University of Pennsylvania immunotherapy specialist who led a previous study on genetic editing, told Nature.
A description of the process Lu’s team employed makes that clear. “The researchers removed immune cells from the recipient’s blood and then disabled a gene in them using CRISPR–Cas9, which combines a DNA-cutting enzyme with a molecular guide that can be programmed to tell the enzyme precisely where to cut,” the Nature article explains.
Carl June is working on a separate trial planned for the US, in which researchers intend to use CRISPR to target three genes in participating patients’ cells. The goal of that study will also be cancer treatment. June hopes that trial will begin in early 2017.
Researchers at Peking University in Beijing are also in the advanced development stages of three clinical trials that will use CRISPR against bladder, prostate, and renal-cell cancers, according to Nature. Those trials are planned for March 2017 but have not yet been approved or secured funding.