The CRISPR craze: genome editing technologies poised to revolutionize medicine and industry

July 22, 2015

Genome editing by engineered Cas9 systems (credit: Mary Ann Liebert, Inc., publishers)

CRISPR/Cas systems for genome editing have revolutionized biological research over the past three years, and their ability to make targeted changes in DNA sequences in living cells with relative ease and affordability is now being applied to clinical medicine and will have a significant impact on advances in drug and other therapies, agriculture, and food products.

The power and promise of this innovation are presented in the Review article “The Bacterial Origins of the CRISPR Genome-Editing Revolution,” published in a special issue of Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available open access until October 15, 2015 on the Human Gene Therapy website.

Erik Sontheimer, University of Massachusetts Medical School, Worcester, and Rodolphe Barrangou, North Carolina State University, Raleigh, describe the origins of this technology, which were derived from DNA sequences found in many bacteria known as clustered, regularly interspaced, short palindromic repeats (CRISPR) regions. These are part of bacteria’s protective immune system.

These regions have been developed into genome editing tools comprised of a “hardware” component (an RNA-guided DNA-targeting system that breaks a DNA strand at a specific site, with the help of the Cas protein), and a “software” component that can be programmed, and re-programmed, to repair or replace a faulty gene.

Impacts beyond academic research

“Although the CRISPR craze has yielded tremendous scientific progress and critical technological advances, it is important to keep in mind that the sgRNA–Cas9 technology is only 3 years old, and that notwithstanding current progress and momentum, we are yet to fully unleash the potential of these tools,” the authors note.

“Beyond academic research and the media, the most significant impact of CRISPR may well turn out to be in industry, with unprecedented levels of interest and investment from multiple distinct business segments, including pharmaceuticals and biotech, as well as covering the food supply chain from agriculture to livestock to other food products.”

Abstract of The Bacterial Origins of the CRISPR Genome-Editing Revolution

Like most of the tools that enable modern life science research, the recent genome-editing revolution has its biological roots in the world of bacteria and archaea. Clustered, regularly interspaced, short palindromic repeats (CRISPR) loci are found in the genomes of many bacteria and most archaea, and underlie an adaptive immune system that protects the host cell against invasive nucleic acids such as viral genomes. In recent years, engineered versions of these systems have enabled efficient DNA targeting in living cells from dozens of species (including humans and other eukaryotes), and the exploitation of the resulting endogenous DNA repair pathways has provided a route to fast, easy, and affordable genome editing. In only three years after RNA-guided DNA cleavage was first harnessed, the ability to edit genomes via simple, user-defined RNA sequences has already revolutionized nearly all areas of biological science. CRISPR-based technologies are now poised to similarly revolutionize many facets of clinical medicine, and even promise to advance the long-term goal of directly editing genomic sequences of patients with inherited disease. In this review, we describe the biological and mechanistic basis for these remarkable immune systems, and how their engineered derivatives are revolutionizing basic and clinical research.