Krishanu Saha, a bioengineer at the University of Wisconsin–Madison whose lab is working on gene therapies for treating blindness, says the precision allowed by CRISPR-Cas9’s programmability is its singular selling point.
“Traditional gene therapy, which we call gene augmentation, is essentially flooding the cell with extra copies of a normal gene; in some cases, this doesn’t work,” Saha tells Inverse. “We found in a few cases, it’s really important to destroy the mutant copy of the [gene] or fix the underlying mutation and that’s where you have to have the precision of CRISPR to go in and specifically do that.”