MULTIPLE GENE EDITS AND COMPUTER SIMULATIONS COULD HELP TREAT RARE GENETIC DISEASES
The lab of Kris Saha at the University of Wisconsin–Madison has developed an innovative combination of gene-editing tools and computational simulations that can be used to develop new strategies for editing genes associated with genetic disorders. In proof-of-concept experiments, the lab’s researchers efficiently corrected multiple mutations responsible for a rare metabolic disorder, known as Pompe disease, in cells …