A new study in mice hints at the potential to use tiny particles made with RNA molecules to deliver chemotherapy drugs and other therapies directly to tumors, killing cancer cells without generating ...

Elevated lipoprotein(a) concentrations are associated with atherosclerotic cardiovascular disease. The safety and efficacy of lepodisiran, an extended-duration, small interfering RNA targeting hepatic ...

CHICAGO -- Novel small interfering RNA (siRNA) agent lepodisiran drastically cut levels of lipoprotein(a), or Lp(a), in the phase II ALPACA trial. The 400-mg dose cut Lp(a) by 93.9 percentage points ...

Editor’s Note: Please see the article for additional information, including other authors, author contributions and affiliations, conflict of interest and financial disclosures, and funding and ...

Small interfering RNA (siRNA) drugs are a class of therapeutic agents that silence specific genes associated with inherited diseases. However, siRNA drugs have challenges because siRNAs often silence ...

The natural RNAi mechanism can be directed by either short interfering RNA (siRNA) or microRNA (miRNA). 1 There are four key steps in the RNAi pathway. When miRNA acts as the guide for RNAi, only a ...

For the first time, a drug to treat adult patients with familial chylomicronemia syndrome (FCS), a severe and rare condition that leads to extremely high levels of blood fats called triglycerides, has ...

Using RNA to interfere with a gene’s expression of disease-causing proteins is a validated therapeutic approach, but so far, the products in this drug class only address liver proteins. Reducing ...