The latest article by Chinese gene editing scholars: "mini" CRISPR genome editing system

"CRISPR can be a simple cutter, or it can be a more advanced regulator, editor, tagger or imager

Many different CRISPR systems are currently used clinically to detect diseases or for gene therapy, such as the eyes, liver, and brain

In a paper published in Molecular Cell on September 3, Professor Qi Lei and others announced an important step forward for CRISPR: an efficient and versatile mini-CRISPR system

Researchers have confirmed in experiments that CasMINI can delete, activate, and edit the genetic code, just like its more powerful counterparts

Sustained effort

To make the system as small as possible, the researchers decided to start with the CRISPR protein Cas12f (also known as Cas14) because it only contains 400 to 700 amino acids

"We thought,'Well, millions of years of evolution have not turned this CRISPR system into something that works in the human body

In fact, Xiaoshu Xu, a postdoctoral scholar in Qi Lei's laboratory and the first author of this paper, did not find the activity of natural Cas12f in human cells

"At first, this system didn't work at all for a year," Xu said.

The result of the first success is not obvious, but it excites them and encourages Xu to continue working hard, because it means the system is successful

"At some point, I had to stop her," Qi Lei recalled, "I said,'It's okay now

In addition to protein engineering, the researchers also designed RNA that guides the Cas protein to the target DNA

Opened the door

Researchers have begun to collaborate with other scientists to seek gene therapy

He said: "From the early days of CRISPR technology, this field needs the ability to design these systems.

Journal Reference :

1. Xiaoshu Xu, Augustine Chemparathy, Leiping Zeng, Hannah R.