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Multiple gene edits and computer simulations could help treat rare genetic diseases – University of Wisconsin-Madison

Research in biomedical engineer Kris Saha’s lab efficiently corrected multiple mutations responsible for Pompe disease, advancing the CRISPR genome-editing field….

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The lab of Kris Saha at the University of WisconsinMadison 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 labs 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…

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