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.
“Can CRISPR fix the problem? The answer is yes. I think we can take the lessons learned from this person and use it to help this person,” Musunuru said.
Musunuru said that a similar one-time fix in humans is potentially helpful not just in extreme cases, but for anyone who suffers a heart attack. Though heart attack survivors typically leave the hospital with a prescription for cholesterol-lowering statins, Musunuru said, within a year less than half are still taking it. A one-time fix via gene therapy before leaving the hospital would likely be more effective.
“The drug doesn’t work if you don’t take it,” said Musunuru, who founded the Cambridge startup Verve Therapeutics to pursue the idea.
Musunuru was among group of experts gathered at the Radcliffe Institute for Advanced Study on Friday to discuss the promise and potential practical and ethical pitfalls posed by the rapid advance of gene therapy owing to CRISPR gene-editing technology.
The all-day event, “Making the Cut: Promises and Challenges of Gene Editing,” brought an audience of several hundred to Radcliffe’s Knafel Center for the institute’s annual science symposium.
Radcliffe’s Life Sciences Adviser, Immaculata De Vivo, highlighted the relatively short history of CRISPR technology, from its discovery as a naturally occurring bacterial immune system in 1987 to scientists’ use of it to precisely cut and edit DNA in 2011, to its application in human cells and, ultimately, its use in living humans, including the recent inauguration of a clinical trial for a treatment for sickle-cell anemia. Along the way, De Vivo said, it has been the enthusiasm and efforts of a global community of researchers that has pushed the technology forward.