Researchers at the University of Georgia have received a $2.78 million grant from the National Institutes of Health to study CRISPR-Cas, a powerful gene editing tool derived from a defense mechanism evolved in bacteria and other single-celled organisms.
Like a pair of molecular scissors, CRISPR-Cas allows scientists to precisely edit sequences of DNA in everything from plants to humans, and it could one day be used to silence the genes that predispose humans to myriad diseases, including cancer, diabetes, cardiovascular disorders and mental illnesses.
“This is a technology that has taken the scientific world by storm,” said Michael Terns, Distinguished Research Professor of Biochemistry and Molecular Biology in UGA’s Franklin College of Arts and Sciences and principal investigator for the project. “But there are still a lot of things that we don’t know, and this project will help us expand and refine our ability to exploit CRISPR for research and biomedicine.”
CRISPR is often discussed as a single entity, but there are actually many different CRISPR-Cas systems, Terns said, and researchers are only beginning to understand the complex molecular processes that make these systems work.
Fundamentally, CRISPR-Cas is a defense mechanism that single-celled organisms use to ward off attacks from viruses and other invaders. For example, when a bacterium is attacked by a virus, it captures some of the virus’s DNA, chops it up into pieces and incorporates a segment of the viral DNA into its own genome.
It then uses this DNA to make RNAs that bind with a protein, and this complex seeks out and destroys the viral DNA.
As the bacterium experiences more threats, it accumulates a bank of past infections in a special part of its genetic code called CRISPRs—short for clustered regularly interspaced short palindromic repeats.