Gene-editing offers patients with genetic disorders hope
The lab of Krishanu 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.
Krishanu Saha, PhD
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 containing the disease-causing errors.
They also used computer simulations to design the ideal gene-editing approach for treating human patients, a boon for rare disorders like Pompe disease that lack useful animal models.