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Researchers Identify Newer And More Precise System For Genome Editing

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An advanced molecular system that can be used as a better pair of genetic scissors for genome editing has been identified by a team of scientists from the McGovern Institute for Brain Research at MIT, Broad Institute of MIT and Harvard, Wageningen University, and National Institutes of Health.

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. At the moment, scientists harness the CRISPR/Cas9 to snip and replace DNA.

In the study published in the journal Cell, CRISPR-Cpf1 is a class 2 CRISPR system that "cleaves DNA via a staggered DNA double-stranded break."

Feng Zhang, lead author of the study, explained that they searched for enzymes that could help engineer human cells. The team discovered two bacterial genomes called Lachnospiraceae and Acidominococcus within Cpf1, which can insert and edit DNA.

The study described the differences between the properties of Cpf1 and Cas9 in terms of how each protein snips and cuts DNA. The Cas9 leaves "blunt ends" within the strands that undergo mutations when rejoined, while the Cpf1 leaves offset cuts that enable short overhangs to be exposed on both ends of the genome. This allows scientists to integrate DNA more accurately.

The researchers have proposed to share the Cpf1 system worldwide through free academic research. Levi Garraway, who is from the Broad Institute but was not involved in the study, said that plans to harness the Cpf1 in cancer research are being made.

In 2014, Zhang, along with the Broad Institute, won the patent for the CRISPR technology despite the fact that research from University of California, Berkeley predates their own. The move sparked a legal battle between the two groups.

Biotech companies have since raced to develop techniques and therapeutics with the CRISPR system.

New York Law School professor Jacob Sherkow says that with Cpf1, a lab can harness the protein without infringing the Cas9 patent. However, it is still uncertain whether the CRISPR/Cpf1 will be patented separately, but Sherkow added that it wouldn't be relevant since it only means that Cas9 is not the only system available for use.

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