In a new study, researchers from the University of Western Ontario in Canada used molecular-Lego to add an engineered enzyme to the revolutionary new gene editing tool, CRISPR/Cas9. Their research suggests that the addition of this enzyme to genes in the targeted genome makes gene editing more efficient and potentially more specific. The relevant research results were published online in the PNAS journal on December 8, 2016, and the title of the paper is "Biasing genome-editing events toward precise length deletions with an RNA-guided TevCas9 dual nuclease".
The scientific community is filled with the promise that CRISPR brings to human genetic editing: it opens the door to the use of gene therapy for diseases such as cystic fibrosis and leukemia.
For example, in cystic fibrosis, the vast majority of patients have a genetic mutation that causes the disease. If this mutation is excised from the genome using CRISPR, the disease may potentially be cured.
"The problem with CRISPR is that it cuts DNA, but then DNA repair removes the incision and pastes it together," says David Edgell, an associate professor of the paper and an associate professor at the University of Western Ontario's Schulich School of Medicine and Dentistry. Regenerating the site that CRISPR is trying to target, creating an inefficient loop. The novelty of adding this engineered enzyme is that it prevents this regeneration from happening."
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