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Gene edited monkeys offer hope for heart disease patients

Meganucleases were used to disable a gene in the livers of rhesus macaques. LAGUNA DESIGN/SCIENCE SOURCE

For the first time, researchers have used gene-editing tools in adult monkeys to disable a gene throughout much of the liver. The approach lowered blood cholesterol levels, suggesting a treatment for heart disease. The study could also pave the way for treating certain genetic diseases caused by a defective, havoc-causing protein.

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

Gene edited primates are nothing new. China has used the famous CRISPR DNA scissors, which snip DNA at a specific location, in monkey embryos to produce animals with modified genomes for studying diseases. More controversially, researchers there have repaired a disease-causing gene in early human embryos with CRISPR, though the embryos were not allowed to develop. And Sangamo Therapeutics, a company in Richmond, California, has employed an older gene-editing tool called zinc finger nucleases to knock out a gene in some of the cells of HIV patients to help them resist the virus. That treatment is known as “ex vivo” because it involves editing a patient’s blood cells in a dish, then putting the cells back into the patient.

Physicians and researchers, however, dream of delivering CRISPR and other genome editors directly into patients to correct mutated genes or treat diseases in other ways. Sangamo, for example, has launched a small clinical trial testing in vivo—into the body—delivery of its nucleases to guide a new gene to a specific location in a small fraction of a patient’s liver cells so they will crank out a needed protein.

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