Researchers Led by George Church Simplify Powerful CRISP/Cas9 Gene Editing Tool by Developing Interactive Software to Find Guide RNAs Predicted to be Highly Specific and Highly Active for Their Gene Targets

Researchers at Harvard University and the University of California, San Diego (UC San Diego), have developed a new user-friendly resource to accompany the powerful gene editing tool called CRISPR/Cas9, which has been widely adopted to make precise, targeted changes in DNA. This new breakthrough has the potential to facilitate new discoveries in gene therapies and basic genetics research. The research was published online on July 13, 2015 in Nature Methods. The article is titled “Unraveling CRISPR-Cas9 Genome Engineering Parameters via a Library-on-Library Approach.” The study describes a way to simplify a laborious part of the gene-editing process using the CRISPR/Cas9 system: choosing the best components to match specific gene targets. "We've taken a step towards making the CRISPR/Cas9 system more robust," said Dr. Prashant Mali, an Assistant Professor in the Department of Bioengineering at the UC San Diego Jacobs School of Engineering, and a co-first author of the Nature Methods publication. CRISPR/Cas9 is a relatively new genome engineering tool that can target a particular segment of DNA in living cells -- such as a gene mutation -- and replace it with a new genetic sequence. This technology ultimately has applications in gene therapies for genetic disorders such as sickle cell anemia and cystic fibrosis. The CRISPR/Cas9 system has two components: a short "guide RNA" with a sequence matching a particular gene target, and a large protein called Cas9 that cuts DNA precisely at that target. Herein lies the beauty of the CRISPR/Cas9 system: to target another region of the genome, researchers can simply change the guide RNA sequence to match the new gene target. However, finding the best guide RNA match for a specific gene target is a labor-intensive process.
Login Or Register To Read Full Story