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URMC / Research / Research@URMC / June 2016 / Review: Giving Gene Editing Technology CRISPR-Cas9 a Boost

Review: Giving Gene Editing Technology CRISPR-Cas9 a Boost

A new gene editing technology called CRISPR-Cas9 has taken the scientific world by storm. It allows researchers to quickly and easily make changes to the DNA of humans, animals and plants. The hope is that CRISPR-Cas9 may be used in the future to eliminate or correct faulty genes that cause disease.DNA

In a recent issue of the journal Cell, Lynne E. Maquat, Ph.D. and Maximilian W. Popp, Ph.D. of the University of Rochester Center for RNA Biology describe how scientists can make this technology more efficient. Understanding the principles of nonsense-mediated mRNA decay (NMD), a cellular mechanism that Maquat discovered early in her career, will help anyone employing the technology achieve a better result – namely, a more complete knock out or deletion of a desired gene.

To use CRISPR-Cas9, scientists pick a gene that they want to remove and program a molecule called a “guide RNA” to go to the site of the gene. The guide RNA carries with it an enzyme called Cas9 that, upon arrival to the site, cuts the DNA that makes up the gene. Typically, the cut causes enough damage to disable the gene, but not always; sometimes the gene is still active and can create proteins that carry out specific functions.  

Lynne E. MaquatMaquat, founding director of the Center for RNA Biology, says knowing how NMD works can help scientists choose where in the gene to make the most beneficial cut. NMD gets rid of messenger RNA (mRNA), which is the template that DNA uses to produce proteins. Cutting DNA in certain spots triggers NMD and eliminates mRNA, robbing DNA of its ability to create functional proteins.

“DNA cuts in locations that generate NMD will be superior to other cuts because they’ll decrease the abundance of mRNA, which limits protein production and increases the likelihood of a complete gene knock out,” noted Maquat, the J. Lowell Orbison Endowed Chair and Professor of Biochemistry and Biophysics at the University of Rochester School of Medicine and Dentistry and an internationally recognized expert in the field of RNA biology. A gene is useless if the DNA that makes up the gene can’t produce any protein.

Maximilian W. PoppThe CRISPR-Cas9 technology is promising, but scientists caution that there is a long way to go before it can be deployed safely and efficiently in people. Popp, a research assistant professor of Biochemistry and Biophysics who works closely with Maquat, says that NMD is one of many tools that can help researchers use the gene editing technique with more precision.

Read the full commentary here.

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The University of Rochester Medical Center is home to approximately 3,000 individuals who conduct research on everything from cancer and heart disease to Parkinson’s, pandemic influenza and autism. Spread across many centers, institutes and labs, our scientists have developed therapies that have improved human health locally, in the region and across the globe. To learn more, visit www.urmc.rochester.edu/research

Emily Boynton | 6/16/2016

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