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Power and Pitfalls of Gene Editing

By October 30, 2019No Comments

Can Anna Feurer’s genes be used to help 10-year-old Avery Watts?

Born with a genetic condition that results in life-threateningly high cholesterol, Watts travels twice a month to a Delaware hospital to have cholesterol filtered from her blood. Feurer, meanwhile, made headlines two years ago when scientists identified a mutation that protects her from heart disease by dramatically lowering levels of blood fats: triglycerides and LDL and HDL cholesterol.

Kiran Musunuru, a cardiologist and associate professor at the University of Pennsylvania’s Perelman School of Medicine, thinks gene therapy can help people like Watts. In recent experiments on rats bred to mimic Watts’ condition, Musunuru used CRISPR-Cas9 gene-editing technology to transfer into them an analog of Feurer’s cholesterol-lowering mutation. After the procedure, the rats’ blood cleared up within days.