New technique developed by UAlberta researchers vastly improves precision of gene-editing technology.
Imagine a future where a guided biomachine put into your body seeks out defective gene sequences in each cell and edits in the correct information with precision accuracy.
It’s called gene editing, and University of Alberta researchers have just published a game-changing study that promises to bring the technology much closer to therapeutic reality.
“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,” said Basil Hubbard, Canada Research Chair in Molecular Therapeutics and an assistant professor in the U of A’s Department of Pharmacology, who led the study.
He and his team have filed a patent on their discovery and are hoping to partner with the pharmaceutical industry to incorporate it into a therapeutic.
Interest in gene-editing technology has been rapidly rising since the discovery of CRISPR/Cas9. This system is naturally present in bacteria, which use it for protection against their natural predators, called 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,” explained Hubbard.
“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.”
Using its natural RNA guide molecule, the Cas9 system is quite accurate, only making a mistake about one per cent of the time, he noted.
“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. One wrong cut and a patient could end up with a serious condition like cancer.”
The new BNA guide molecule that Hubbard and his team—which includes PhD student Christopher Cromwell, who is first author on the study—developed was shown to be much more stable and stringent in its quest for finding the right DNA to cut.
“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,” said Hubbard.
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 hemophilia and various cancers.
The Latest on: Gene editing
via Google News
The Latest on: Gene editing
Swift Gene-Editing Method May Revolutionize Treatments for Cancer and Infectious Diseases
on July 11, 2018 at 10:31 am
A colored scanning electron micrograph of a white blood cell amid red blood cells. Researchers hope a new method to boost immune systems could make a treatment available to patients with almost any ty... […]
T cell engineering breakthrough sidesteps need for viruses in gene-editing
on July 11, 2018 at 10:02 am
Scanning electron micrograph of a human T lymphocyte (also called a T cell) from the immune system of a healthy donor. Credit: NIAID In an achievement that has significant implications for research, m... […]
Researchers discover why CRISPR gene editing sometimes fails
on July 11, 2018 at 8:45 am
A study conducted at the University of Illinois, Chicago has explained for the first time why the CRISPR gene editing tool sometimes fails to work. The study authors also suggest steps that can be tak... […]
Researchers reveal why CRISPR gene editing sometimes fails to work
on July 11, 2018 at 3:01 am
Researchers from the University of Illinois at Chicago are the first to describe why CRISPR gene editing sometimes fails to work, and how the process can be made to be much more efficient. CRISPR is a ... […]
Ag scientists draw a road map for gene editing
on July 11, 2018 at 2:42 am
To consumers trying to get their heads around what’s good or bad about the new crop and animal gene-editing techniques, and to scientists and farm policy stalwarts racing to stay abreast of that front... […]
Biochemists discover cause of genome editing failures with hyped CRISPR system
on July 10, 2018 at 11:49 pm
Credit: Ryan Clarke, et al Researchers from the University of Illinois at Chicago are the first to describe why CRISPR gene editing sometimes fails to work, and how the process can be made to be much ... […]
Reducing Cholesterol with Gene Editing
on July 10, 2018 at 4:10 pm
Scientists know that there is a link between a protein called PCSK9 and cholesterol; when the protein is not active, cholesterol levels go down. Therapies that aim to use this relationship to the pati... […]
Fetal Gene Editing with Synthetic Nucleotides Cures Beta-Thalassemia in Mice
on July 10, 2018 at 7:02 am
Scientists at Yale University and Carnegie Mellon University have used gene editing to effectively cure the genetic blood disorder β-thalassemia, in mouse fetuses, in utero. The approach, which harnes... […]
Gene editing successfully lowers monkey cholesterol levels
on July 9, 2018 at 7:43 pm
Researchers from the University of Pennsylvania have lowered the blood cholesterol in monkeys using gene editing(Credit: andriano_cz/Depositphotos) In one of the first examples of gene-editing technol... […]
via Bing News