Researchers at the University of Alberta (UAlberta) have devised a new method to significantly increase the accuracy of gene-editing technology, taking it ‘one step closer’ to application in patients.
Gene editing involves correcting information on defective gene sequences in the body.
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It has been previously discovered that the CRISPR / Cas9 system, which is naturally used by bacteria to protect from predators, can be altered to edit human genes by cutting a select DNA sequence. The system is driven by a natural RNA guide molecule.
The new technique seeks to replace the natural guide molecule with a synthetic molecule called bridged nucleic acid (BNA).
UAlberta Pharmacology department assistant professor Basil Hubbard said: “One of the main issues is that the system is not perfectly specific – sometimes it cuts a similar but incorrect gene.
“However, given that there are trillions of cells in the human body, even one percentage off is quite significant, especially because gene editing is permanent.
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By GlobalData“One wrong cut and a patient could end up with a serious condition like cancer.”
The team reported that the new BNA guide molecule is more stable and stringent in finding the correct DNA to cut.
Hubbard noted: “Our research shows that the use of bridged nucleic acids to guide Cas9 can improve its specificity by over 10,000 times in certain instances, a dramatic improvement.”
Though gene-editing technology still has several hurdles to overcome, including the challenge of how to deliver it effectively into the human body, it may someday be used to treat a wide variety of genetic diseases, from muscular dystrophy to haemophilia and various cancers.
