Scientists solved structure of new protein to be used for genome editing

MOSCOW, May 17. /TASS/. An international group of scientists have solved the crystal structure of the Cpf1 protein. This protein is part of a CRISPR system has potential for use in genome editing and may have advantages over popular editing tools based on the Cas9 protein, Skotech press service said.

CRISPR-Cas9 technology is revolutionizing the field of genomics. "CRISPR" stands for Clustered Regularly Interspaced Short Palindromic Repeats, which are key components of a system used by bacteria to defend against viruses. Cas9 - one of the enzymes produced by the CRISPR system - binds to DNA in a highly sequence-specific manner and cuts it, allowing researchers to target specific regions of the genome and edit them, curing mutations. The new study will serve as a foundation for development of a similar system based on CRISPR-Cpf1.

"The Cpf1 protein is a single effector protein that was found in Acidaminococcus sp. It efficiently creates double-stranded breaks in DNA and has a simpler way of function compared to the classical Cas9 CRISPR effector. Cpf1 can be used for genome editing, activation of gene transcription, or targeting DNA sequences for visualization by microscopy. Making such tools requires understanding of how the protein recognizes and cleaves target DNA. The structural information described in our paper will facilitate the development of a future CRISPR-Cpf1 toolbox", said Skoltech PhD student Yana Fedorova, supervised by Professor Konstantin Severinov.

This work is part of a long-term collaboration between Skoltech DIBB, Eugene Koonin's lab at the National Institutes of Health (Bethesda, USA) and Feng Zhang’s lab at the Massachusetts Institute of Technology (Boston, USA). Their goal is to systematically search for new CRISPR systems by means of bioinformatics and check the bioinformatic predictions experimentally. Results of the work were published in journal Cell.