Gene editing progress with the new CRISPR / Cas9 variant

Using a new variant to repair DNA will improve both the safety and efficacy of the much-touted CRISPR-Cas9 tool in genetic research, say Michigan Medicine researchers.

These two key issues, safety and efficacy, are what continue to hold the CRISPR-Cas9 gene back from its full clinical potential, explains co-senior author Y. Eugene Chen, MD, Ph.D., professor of internal medicine, cardiology surgery, physiology, pharmacology and medicinal chemistry, from the Michigan Medicine Frankel Cardiovascular Center.

The new CRISPR-Cas9 variant improves efficiency when inserting a gene or DNA fragment at a precise location in the genome, known as knocking in. It also reduces the rate of unwanted insertions or deletions, known as indel, of base pairs that often occur gene editing.

“We call it meticulous integration Cas9, or miCas9, to reflect its extraordinary ability to allow maximum integration, but with minimal indels, as well as to recognize its development at the University of Michigan,” write senior authors Chen, Jifeng Zhang and Jie Xu for Naturefrom the “Behind the Paper” series. “Provides a” one small stone for three birds “tool in gene editing”.

The team previously reported Cas9 genome editing in 2014 and reported the beneficial effects of a RAD51 agonist, RS-1, in gene editing in 2016.