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ma=86400 RNA gene therapies offer hope for millions with high cholesterol
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RNA gene therapies offer hope for millions with high cholesterol

Base editing, antisense oligonucleotides and siRNA therapies that target low-density lipoprotein and the genes encoding molecules that regulate it are being tested in several clinical trials.
  1. Natalie Healey

    1. Natalie Healey is a freelance writer in London, UK.

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Image credit: Denis Pobytov / DigitalVision Vectors / Getty

Since the late 1980s, statins have been the mainstay of cholesterol management, lowering low-density lipoprotein (LDL) levels and preventing countless heart attacks. Yet many patients do not achieve their target LDL cholesterol levels, either because adherence to daily medication is a challenge or because their genetic profiles make achieving optimal cholesterol levels more difficult. Emerging RNA-based therapies, however, are changing the treatment landscape by targeting cholesterol at the genetic level in the liver (where most cholesterol is made), offering the possibility of long-lasting effects with fewer doses. Although these drugs were initially developed for people with genetic causes of high cholesterol, clinical trials are now testing their wider use.

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doi: https://doi.org/10.1038/d41591-025-00002-2

The Clinical Pipeline is a column on translational and clinical research, from bench to bedside.

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