Genredigerande läkemedel sänker det onda kolesterolet med 62 procent i studie

En experimentell terapi vid namn VERVE-102 sänkte LDL-kolesterolet med upp till 62 procent efter en enstaka dos i en tidig säkerhetsstudie. Resultaten kommer från en fas I-studie med 35 patienter som lider av högt kolesterol eller tidig hjärt-kärlsjukdom. Data publicerades denna vecka i New England Journal of Medicine.

Forskare rapporterade att behandlingen verkade säker, utan några allvarliga biverkningar ens vid den högsta dosen. Deltagarna upplevde endast en tillfällig mild ökning av leverenzymer. Läkemedlet använder mRNA förpackat i nanopartiklar för att redigera PCSK9-genen i leverceller, vilket permanent minskar produktionen av ett protein som höjer LDL-nivåerna.

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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.

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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.

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