New Genome Editing System Is a Revolution in Human Medicine

They are said to help the bacteria in the body to fight against the viral attacks.

The CRISPR/Cas9 system evolved as a way for bacteria and archaea to defend themselves against invading viruses. The bacteria in her samples were observed carrying out Cas9 proteins, which were able to cut the DNA based on an RNA guide sequence with great precision. The new technique has already been used by scientists for engineering crops, livestock and human embryos. They found two, from Acidominococcus and Lachnospiraceae, that can snip DNA when scientists insert them into human cells.

“We have been thrilled to find utterly totally different CRISPR enzymes that may be harnessed for advancing analysis and human well being”, stated Feng Zhang of Broad Institute. It has huge implications for therapies and research, and could be a turning point in many businesses and intellectual property cases.

In its natural form, the DNA-cutting enzyme Cas9 forms a complex with two small RNAs, both of which are required for the cutting activity. The Cpf1 system is simpler in that it requires only a single RNA. Cpf1 is also smaller than the Cas9 protein, making it easier to deliver into cells and tissues.

Second and perhaps the most important is the way in which Cpf1 cuts the DNA. When Cas9 cuts DNA, it splits both strands of the molecule in at the same place, leaving blunt ends behind that are subject to mutations upon being reconnected. With the Cpf1 complex the cuts in the two strands are offset, leaving short overhangs on the exposed ends. “It makes the insertion much more controllable”. This allows scientists to integrate DNA more accurately. It also leaves extended parts on the cut side, so if targeted genome becomes mutated at a side, it can still be re-cut and corrected multiple times.

The Cpf1 system provides new flexibility in choosing target sites. The microbiologists noticed odd repeating sequences in the DNA of bacteria in the 1980s, and those clustered regularly interspaced short palindromic repeats turned into CRISPR. This could be an advantage in targeting some genomes, such as in the malaria parasite as well as in humans.

“This has dramatic potential for advance genetic engineering”, Eric Lander, the director of Broad Institute of MIT and Harvard and one of the leaders of the project.

The workforce together with the scientist who first harnessed the revolutionary CRISPR-Cas9 system for mammalian genome editing, described the sudden organic options of the brand new system and demonstrated that it may be engineered to edit the genomes of human cells. Identifying this mechanism of interference broadens our understanding of CRISPR-Cas systems and advances their genome editing applications.

“The unexpected properties of Cpf1 and more precise editing open the door to all sorts of applications, including in cancer research”.

Zhang, Broad Institute, and MIT plan to share the Cpf1 system widely.

“The more [CRISPR] systems the better”, said biologist Kate O’Connor-Giles of the University of Wisconsin, Madison, who uses the tool to study brain development and was not involved in the new study.

The researchers discovered that the biological features of Cpf1, a protein that is different from the CRISPR/Cas9, has the potential to improve genetic engineering since its features are simpler and more powerful.

The question on everyone’s minds now is, will this new enzyme surpass Cas9 in popularity?