"There was a need in the community to have a technique that you could use to generate targeted mutations," says Jill Wildonger, a UWMadison assistant professor of biochemistry and another senior author of the paper. "The need was there and this was the technical advance that everyone had been waiting for."
"The reason this has progressed so quickly is that many researchers us included were working on other, more complicated, approaches to do exactly the same thing when this came out," adds genetics assistant professor Kate O'Connor-Giles, the third senior author. "This is invaluable for anyone wanting to study gene function in any organism and it is also likely to be transferable to the clinical realm and gene therapy."
The CRISPR RNA/Cas9 system directs a DNA-clipping enzyme called Cas9 to snip the DNA at a targeted sequence. This cut then stimulates the cell's existing DNA repair machinery to fill in the break while integrating the desired genetic tweaks. The process can be tailored to edit down to the level of a single base pair the rough equivalent of changing a single letter in a document with a word processor.
The broad applicability of the system is aided by a relatively simple design that can be customized through creation of a short RNA sequence to target a specific sequence in the genome to generate the desired changes. Previous genome editing methods have relied on making custom proteins, which is costly and slow.
"This is so readily transferable that it's highly likely to enable gene knockout and other genome modifications in any organism," including those that have not previously been used for laboratory work, says O'Connor-Giles. "It's going to turn non-model organisms into genetic model organisms."
|Contact: Melissa Harrison|
University of Wisconsin-Madison