Obat penyunting gen pangkas kolesterol jahat hingga 62 persen dalam uji klinis

Terapi eksperimental bernama VERVE-102 berhasil menurunkan kolesterol LDL hingga 62 persen setelah satu dosis tunggal dalam studi keamanan tahap awal. Hasil ini berasal dari uji klinis Fase I yang melibatkan 35 pasien dengan kolesterol tinggi atau penyakit kardiovaskular dini. Data tersebut diterbitkan minggu ini di New England Journal of Medicine.

Para peneliti melaporkan bahwa perawatan ini tampak aman, tanpa efek samping serius bahkan pada dosis tertinggi. Partisipan hanya mengalami peningkatan ringan sementara pada enzim hati. Obat ini menggunakan mRNA yang dikemas dalam nanopartikel untuk menyunting gen PCSK9 di sel hati, sehingga secara permanen mengurangi produksi protein yang meningkatkan kadar LDL.

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Scientific illustration of HELZ2 protein in the liver regulating cholesterol release.
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UT Southwestern researchers identify HELZ2 protein that controls the liver’s release of cholesterol-carrying particles

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Scientists at UT Southwestern Medical Center report they have identified a protein, HELZ2, that acts as a key regulator of how many cholesterol-carrying particles the liver releases into the bloodstream by affecting the gene APOB. The study was published in the American Heart Association journal Circulation and could inform future research into heart disease and fatty liver disease.

An experimental oral pill called enlicitide lowered LDL cholesterol by about 60% in a large phase three clinical trial, according to results published in The New England Journal of Medicine. The trial, led by Dr. Ann Marie Navar at UT Southwestern Medical Center and sponsored by Merck, involved 2,909 participants mostly already on statins. If approved, the daily pill could improve access to effective cholesterol treatment.

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Researchers from the University of Barcelona and the University of Oregon report that short DNA molecules known as polypurine reverse Hoogsteen hairpins (PPRHs) suppressed the PCSK9 gene and reduced blood cholesterol in a mouse model. In transgenic mice carrying the human PCSK9 gene, a single injection of one candidate (HpE12) cut plasma PCSK9 by 50% and total cholesterol by 47% three days later, according to findings published in Biochemical Pharmacology.

Researchers in Australia have found that a mysterious FDA-approved drug, called compound X, removes toxic alpha-synuclein proteins from the brains of mice with Parkinson's-like symptoms. The treatment improved the animals' balance and mobility by enhancing the brain's glymphatic waste disposal system. The findings were presented at a symposium in the UK.

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A large review of cardiovascular outcome trials found that people taking GLP-1 receptor agonists—drugs that include semaglutide (sold as Ozempic)—had a lower risk of major heart-related events than those given placebo. The analysis pooled results from 11 trials involving more than 90,000 participants, with an average follow-up of nearly three years, and reported benefits across patient subgroups including those with and without diabetes.

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