A foundation for future research
Since its discovery, researchers have hailed Cas9 — a protein “machine” that can be programmed by a strand of RNA to target specific DNA sequences and to precisely cut, paste, and turn on or turn off genes — as a potential key to unlocking a host of new treatments and therapies for genetic conditions, but only if they fully understand how it works. That’s where David R. Liu and his students Vikram Pattanayak and John Guilinger come in. Liu, a Harvard professor of chemistry and chemical biology and an investigator with the Howard Hughes Medical Institute, joined with Professor Jennifer Doudna of the University of California, Berkeley, to lead an effort to develop a detailed “specificity profile” for Cas9 — data to reveal how accurately Cas9 can home in on the DNA sequence it is programmed to target, and how susceptible the protein/RNA complex is to acting on decoy off-target sequences instead....