Menu

ISTOCK, ROBBIE ROSS

Gene Editing Reduces Monkeys’ Cholesterol

The results could lead to a treatment to lower cholesterol in patients with hypercholesterolemia.

Jul 10, 2018
Ashley Yeager

Editing monkeys’ genomes in their livers reduced the animals’ blood cholesterol levels, researchers reported yesterday (July 9) in Nature Biotechnology. The results suggest the technique could one day be used to treat certain heart disease patients who do not tolerate drugs designed to combat high cholesterol. 

“It’s very nice work, one of the first demonstrations of gene-editing tools used with high efficiency in nonhuman primates,” Kiran Musunuru, a cardiologist and geneticist at the University of Pennsylvania who was not involved in the study, tells Science.

In the study, University of Pennsylvania gene therapy researcher James Wilson and his colleagues used a gene-editing tool called a meganuclease to target and inactivate the gene PCSK9, which produces a protein that prevents the body from removing LDL, the “bad” form of cholesterol, in the monkeys’ livers. The approach “worked incredibly well,” Wilson tells Science

PCSK9 levels dropped by as much as 84 percent and LDL levels dipped as much as 60 percent in treated monkeys, the researchers report in the paper. The team now plans to work on preventing editing in sites other than the PCSK9 gene and any unwanted immune responses to the treatment so that it could be moved into clinical trials in humans to test its efficacy in patients with hypercholesterolemia, who don’t tolerate current cholesterol-lowering drugs that inhibit the PCSK9 protein. 

“Most often these patients are treated with repeated injections of an antibody to PCSK9,” study coauthor Lili Wang says in a statement. “But, our study shows that with successful genome editing, patients who cannot tolerate inhibitor drugs might no longer need this type of repeat treatment.”

Many gene-editing tools are currently being tested, however, so “it’s too early to tell which of those approaches will translate” into people, MIT molecular geneticist Daniel Anderson tells Science.

February 2019

Big Storms Brewing

Can forests weather more major hurricanes?

Marketplace

Sponsored Product Updates

Bio-Rad Showcases New Automation Features of its ZE5 Cell Analyzer at SLAS 2019
Bio-Rad Showcases New Automation Features of its ZE5 Cell Analyzer at SLAS 2019
Bio-Rad Laboratories, Inc. (NYSE: BIO and BIOb) today showcases new automation features of its ZE5 Cell Analyzer during the Society for Laboratory Automation and Screening 2019 International Conference and Exhibition (SLAS) in Washington, D.C., February 2–6. These capabilities enable the ZE5 to be used for high-throughput flow cytometry in biomarker discovery and phenotypic screening.
Andrew Alliance and Sartorius Collaborate to Provide Software-Connected Pipettes for Life Science Research
Andrew Alliance and Sartorius Collaborate to Provide Software-Connected Pipettes for Life Science Research
Researchers to benefit from an innovative software-connected pipetting system, bringing improved reproducibility and traceability of experiments to life-science laboratories.
Corning Life Sciences to Feature 3D Cell Culture Technologies at SLAS 2019
Corning Life Sciences to Feature 3D Cell Culture Technologies at SLAS 2019
Corning Incorporated (NYSE: GLW) will showcase advanced 3D cell culture technologies and workflow solutions for spheroids, organoids, tissue models, and applications including ADME/toxicology at the Society for Laboratory Automation and Screening (SLAS) conference, Feb. 2-6 in Washington, D.C.
Corning Introduces New 1536-well Spheroid Microplate
Corning Introduces New 1536-well Spheroid Microplate
High-throughput spheroid microplate benefits cancer research, drug screening