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    Home > Biochemistry News > Biotechnology News > A treatment that reduced bad cholesterol by 61% In animal experiments with gene editing therapy showed lasting results

    A treatment that reduced bad cholesterol by 61% In animal experiments with gene editing therapy showed lasting results

    • Last Update: 2021-01-22
    • Source: Internet
    • Author: User
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    HeFH affects about one in every 200 to 500 people worldwide.
    patients usually carry a mutation in the LDLR gene, which causes a decrease in LDLR expression and elevates LDL-C levels in the blood.
    over time, high LDL-C accumulates in the arteries of the heart, causing blood flow to decrease or block, eventually leading to a heart attack or stroke.
    previous studies have shown that PCSK9 gene infestion can increase LDL-patient expression, leading to lower LDL-C levels and reducing the risk of coronary heart disease.
    VERVE-101 causes the PCSK9 gene to inferces by altering the DNA genetic sequence of PCSK9 by a single A-to-G.
    latest data have shown that monobase editing of the PCSK9 gene in the livers of non-human primates can consistently reduce levels of LDL-C in the blood and PCSK9 proteins in the blood.
    study, a single intravenous infusion reduced blood LDL-C by 59% in week 2 and was maintained within 6 months of treatment.
    LDL-C decreased by an average of 61% over that time period.
    the average blood PCSK9 protein level decreased by 89% over the same six-month period.
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