You have an immune system. Your cat has an immune system. And bacteria have an immune system, too—one that we’ve tapped to make one of the most powerful tools ever for editing genes.
The tool is called CRISPR (for “clustered regularly interspaced short palindromic repeats”), and it makes use of enzymes that “remember” viral genes and cut them out of bacterial genomes. Applied to bioengineering, CRISPR is launching a revolution. And the Boston Globe reported over the weekend that while researchers at the University of California at Berkeley first developed CRISPR, the technique is booming in labs around Boston.
Local researchers have founded two companies aimed at turning the technique into a therapy to fix errant genes that cause a range of illnesses. Meanwhile, almost every week the method seems to be used for a new purpose: MIT researchers have used it to rapidly engineer mice with liver tumors; Harvard researchers have used it to disrupt genes and lower cholesterol levels in mice; and [Boston Children’s Hospital] scientists have, using human stem cells, corrected gene mutations that cause a rare blood disorder.
As the Globe noted, CRISPR has quickly become a popular tool at Boston Children’s Hospital, too. Just last month Derrick Rossi, PhD, of our Program in Cellular and Molecular Medicine, and his colleague Chad Cowan, PhD, of Massachusetts General Hospital, announced that they’d worked out a way to use CRISPR to edit genes out of blood stem cells—a first in the technique’s brief history. George Q. Daley, MD, PhD, of our Stem Cell Program, is testing whether it can correct sickle cell disease (SCD), building on induced pluripotent stem cell (iPSC) research and basic work on the biology of SCD by Stuart Orkin, MD, of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.
Check out this CRISPR primer by Vector contributor Nicole Davis, which lays out how the technique works and its potential impact on disease research and therapeutic development.