CRISPR genetic editing methods in target diseases

The CRISPR revolution has begun and shows no signs of slowing down. This system, which is the key to prokaryotic adaptive immunity, has proven to be particularly under the influence of genome engineering. CRISPR/Cas provides flexibility as well as easily amplifying the signal and regulating the signal level, which is beyond the capabilities of previous systems.

Prokaryotes have long exploited CRISPR/Cas as a powerful defense strategy against virus-derived invaders, and eventually, this system turns out to be useful only for research applications. Just when we thought that genome engineering could not get any further, the CRISPR (clustered regularly spaced short palindromic repeats)/Cas9 system emerged. It is an important component of the bacterial immune system, allowing bacteria to recognize and destroy phages.

In genome engineering applications, guide RNA (gRNA) sequence homology targets the Cas9 endonuclease to a specific locus-where the double helix is ​​broken. Like ZFNs and TALENs, CRISPR/Cas9 uses HDR, but using RNA to determine editing makes the system more sensitive and malleable, as well as less expensive and time-consuming. For this reason, CRISPR/Cas9 has proven invaluable for high-throughput genome engineering.

With a variety of robust and effective genome engineering methods at our fingertips, we have entered the Golden Age of genome engineering. Current studies have focused on developing these techniques in the hope that they will be clinically useful to provide desired target loci (or loci), high specificity, and efficacy. The most exciting thing we've found at Addgene is the democratization of genome engineering that has enabled and will continue to enable researchers around the world to use these tools.