New Gene Editing Technology Has Vast Implications

[Cort]

Another example of science moving forward at incredible speed. Researchers can already apparently alter gene polymorphisms so that they're no longer damaging (the COMT and complement genes recently came up big in ME/CFS). This finding could allow researchers to alter the expression of any gene. They're not altering the gene - they're just turning it off.

If gene expression studies identify key genes at work in ME/CFS or FM - this technology could help turn them off....

It's a brave new world indeed.

• https://www.urmc.rochester.edu/rese...-editing-technology-holds-huge-potential.aspx

Gene Editing Technology Holds Huge Potential
A new gene editing technology known as CRISPR-Cas9 is taking the scientific community by storm. It is based on a system that bacteria use to defend themselves and their descendants against viruses that can make them sick (yes, bacteria can get sick). Over the last few years, researchers have used this system to precisely alter protein coding genes (genes that provide instructions for making proteins) in human cells, mice, rats, zebrafish, fruit flies and plants. In some cases, disease causing mutations in a protein have been corrected with CRISPR-Cas9, offering some hope for therapeutic intervention.

Now, for the first time, researchers from the Aab Cardiovascular Research Institute (CVRI) at the University of Rochester have used CRISPR-Cas9 to change a regulatory element – a non-protein coding snippet of DNA that controls a gene’s expression – in the laboratory mouse.

“To our surprise, we found that by making a very subtle change in DNA using the CRISPR-Cas9 technology we were able to virtually wipe out the expression of a gene,” said lead study author Joseph Miano, Ph.D., associate professor and associate director of the CVRI. “With this revolutionary technology scientists can literally edit any nucleotide in the 3 billion-plus nucleotides that make up the blueprint of mammalian life forms, including humans.”

According to Miano, the study of these snippets of DNA is extremely relevant because most disease-causing mutations occur in the 98.5 percent of the human genome that does not code for proteins. He says that what was once considered junk DNA is in fact replete with non-coding genes and millions of snippets of DNA that dictate if and when genes are turned on or off and for how long. He says we can now easily model variations in key regulatory snippets that occur in humans predisposed to certain diseases. His study was published earlier this year in the journal Arteriosclerosis, Thrombosis and Vascular Biology.