CRISPR corrected a cataract-causing genetic defect in a mutant mouse (left); control cataract-model mouse (right).JINSONG LIIt was less than a year ago that scientists first applied CRISPR, a genome-editing technique, to human cells. In short order, the technique has taken off like wildfire. And now, two papers appearing in Cell Stem Cell today (December 5) show that CRISPR can be used to rewrite genetic defects to effectively cure diseases in mice and human stem cells.
“What’s significant about this is it’s taking CRISPR to that next step of what it can be used for, and in this case, it’s correcting mutations that cause disease,” said Charles Gersbach, a genomics researcher at Duke University, who was not involved in either study.
CRISPR stands for clustered regularly interspaced short palindromic repeats. These RNA sequences serve an immune function in archaea and bacteria, but in the last year or so, scientists have seized upon them to rewrite genes. The RNA sequence serves as a guide to target a DNA sequence in, say, a zygote or a stem cell. The guide sequence leads an enzyme, Cas9, to the DNA of interest. Cas9 can cut the double strand, nick it, or even knock down gene ...
