Knocking out cholesterol genes could offer ‘one-and-done’ CRISPR cure for heart disease

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Credit: Healthline

When CRISPR “base editing” was used to knock out two cholesterol-associated genes in monkeys, the animals’ blood levels of heart-disease-causing LDL (“bad”) cholesterol and triglycerides plunged as much as 60% and 65%, respectively, Sekar Kathiresan, co-founder and CEO of Verve Therapeutics, announced on [June 27] at the (virtual) meeting of the International Society for Stem Cell Research.

The results, from Verve’s experiments in 14 cynomolgus monkeys (a.k.a. crab-eating macaques), are the first published data showing successful CRISPR base editing in a non-human primate; there have been similar successes in mice. They are therefore good news not only for Verve, which was founded last year to develop CRISPR-based cures for cardiovascular disease, but also for Beam Therapeutics, a two-year-old company developing CRISPR base editors for a long list of diseases. Verve licensed Beam’s “adenine base editor” for its experiment.

Related article:  CRISPR gene-editing can help turn Africa's low-yielding rice varieties into sustainable staple crops

“Our goal is to develop a one-and-done genome editing medicine for heart disease,” Kathiresan told STAT ahead of his ISSCR talk.

“We really do want to transform how we think about cardiovascular disease,” Kathiresan said. Instead of taking statins for decades… people who undergo gene therapy would, if all goes well, have the same protection against heart disease as individuals with natural mutations in PCSK9, ANGPTL3, or six other protective genes in Verve’s sights.

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