Gene-editing drug slashes bad cholesterol 62 percent in trial

An experimental therapy called VERVE-102 lowered LDL cholesterol by up to 62 percent after a single dose in an early safety study. The results come from a Phase I trial involving 35 patients with high cholesterol or early cardiovascular disease. Data were published this week in the New England Journal of Medicine.

Researchers reported that the treatment appeared safe, with no serious side effects even at the highest dose. Participants experienced only a temporary mild rise in liver enzymes. The drug uses mRNA packaged in nanoparticles to edit the PCSK9 gene in liver cells, permanently reducing production of a protein that raises LDL levels.

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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.

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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A new study indicates that measuring apolipoprotein B could help prevent more heart attacks and strokes than the standard LDL cholesterol test used by millions of Americans.

A single mRNA injection can halve the rate of chromosome errors in human eggs from older women, according to new research presented at a London conference. The treatment targets a protein deficiency that contributes to aneuploidy, a common cause of IVF failure and miscarriage. Researchers say the approach shows promise for improving fertility outcomes.

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Researchers from the Institute for Bioengineering of Catalonia and collaborating institutions report that engineered “supramolecular” nanoparticles restored aspects of blood-brain barrier function in Alzheimer’s-model mice, rapidly lowering brain amyloid-β and producing improvements on behavioral and memory tests.

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