Researchers have reportedly developed a new technique that dramatically increases the accuracy of gene-editing technology, bringing it a step closer to patient applications.
In a study published in the journal Natural Sciences by researchers from the University of Alberta in Canada, the team described a new technique that significantly improves the accuracy of current gene-editing technology.
The researchers achieved this by replacing the essential guide molecule used by the gene-editing tech with a synthetic one known as BNA.
“We’ve discovered a way to greatly improve the accuracy of gene-editing technology by replacing the natural guide molecule it uses with a synthetic one called a bridged nucleic acid, or BNA,” Basil Hubbard, an assistant professor in the U of A’s Department of Pharmacology and current Canada Research Chair in Molecular Therapeutics who led the study, said.
Since the discovery of CRISPR-Cas9, the interest of researchers in gene-editing has grown exponentially. The Cas9 system is reportedly present in bacteria which use it as a protection against natural predators referred to as bacteriophages.
“It allows bacteria to store information about previous infections and then use it to seek out and destroy the DNA of new invaders by cutting it,” Hubbard explained.
“What researchers have realized is that this system can be programmed to cut a specific DNA sequence in a human cell also, allowing us to edit our genes. One of the main issues, however, is that the system is not perfectly specific—sometimes it cuts a similar but incorrect gene.”
The Cas9 system has a one percent mistake rate which makes it quite accurate. However, with trillions of cells in a human body, Hubbard said that one percent is still quite significant. That is because gene-editing is permanent.
“One wrong cut and a patient could end up with a serious condition like cancer,” Hubbard added.
The new BNA molecule guide is said to be more stable and stringent when it comes to finding and locating the right DNA to cut. According to the study, using the bridged nucleic acids to guide Cas9 can significantly improve its specificity by more than 10,000 times in certain instances.
The breakthrough study promises to bring the controversial technology closer to applications to patients and therapeutical reality.
Hubbard and his team have already filed a patent for their discovery and are now hoping to collaborate with the pharmaceutical industry for future medical applications.