CRISPR/Cas9 can reverse multiple diseases in mice
New use for the genetic tool turns genes on instead of snipping them
PUMP IT UP A new type of CRISPR/Cas9-based gene therapy turned on muscle-building genes in mice that have muscular dystrophy. Muscle fibers (magenta) from mice that got the therapy (right) were bigger than fibers from untreated animals (left).
H.-K. Liao et al/Cell 2017
A new twist on gene editing makes the CRISPR/Cas9 molecular scissors act as a highlighter for the genetic instruction book. Such highlighting helps turn on specific genes.
Using the new tool, researchers treated mouse versions of type 1 diabetes, kidney injury and Duchenne muscular dystrophy, the team reports December 7 in Cell. The new method may make some types of gene therapy easier and could be a boon for researchers hoping to control gene activity in animals, scientists say.
CRISPR/Cas9 is a two-part molecular scissors. A short, guide RNA leads the DNA-cutting enzyme Cas9 to specific places in the genetic instructions that scientists want to slice. Snipping DNA is the first step to making or fixing mutations. But researchers quickly realized the editing system could be even more versatile.
In the roughly five years since CRISPR/Cas9 was first wielded, researchers have
