• AutoTL;DRB
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    38 months ago

    This is the best summary I could come up with:


    In a small initial test in people, researchers have shown that a single infusion of a novel gene-editing treatment can reduce cholesterol, the fatty substance that clogs and hardens arteries over time.

    This places a very heavy treatment burden on patients, providers, and the health care system,” said Andrew Bellinger, chief scientific officer of Verve Therapeutics, at a news conference over the weekend.

    In a study published in the journal Circulation earlier this year, researchers from the company showed that the approach lowered bad cholesterol 49 to 69 percent in monkeys, depending on the dosage they received.

    While the participants already had severe coronary artery disease, and some had previously experienced a heart attack, the company aims to eventually treat younger patients in order to prevent these outcomes.

    Sanjay Rajagopalan, director of the Cardiovascular Research Institute at Case Western Reserve University School of Medicine, calls the results a “very exciting” proof of concept.

    But Rajagopalan, who wasn’t involved in the Verve study, says the main concern about any Crispr-based approach is the potential for off-target effects, in which unwanted cells or genes would unintentionally be edited.


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