Gene Editing and the Treatment of Hemophilia B
The discovery of the CRISPR/Cas9 system forever changed our world, allowing scientists to quickly and efficiently edit DNA. Of course, we could edit DNA before; however, the methods were not terribly precise. And what is, perhaps, most notable about the CRISPR system is that it allows scientists to make their edits at a reasonable price. And in a world where scientific breakthroughs are largely determined by whether or not one is able to get funding for their research, the significance of affordable techniques simply cannot be overstated.
So how does it work? In short, Cas9 is one of the enzymes produced by the CRISPR system. It binds to DNA in a highly sequence-specific manner and cuts it, allowing for accurate DNA manipulation.
With this technique, gene editing has truly taken off. It is highly more efficient than the previous mainstream methods that make use of TAL Effector Nucleases (TALENS) and Zinc Finger Nucleases (ZFNs), and the new method paves way for more researches and, possibly, using the use of gene editing to treat human illnesses and ailments.
In fact, this is precisely what researchers from Richmond, California’s Sangamo BioSciences are trying to do.