Related Experiment Video
Updated: Mar 2, 2026
09:15
Differential Effects of Lipid-lowering Drugs in Modulating Morphology of Cholesterol Particles
Published on: November 10, 2017
15.2K
RNA interference for lipid disorders: is this the future?
Miguel Nassif1, Renata Turrini Jacob Fazoli1, Giuliano Generoso1,2
1Cardiology Center, Hospital Sirio Libanes.
Current Opinion in Lipidology
|February 28, 2026
Summary
RNA interference (RNAi) therapies, using small interfering RNA (siRNA) drugs, offer targeted gene silencing for lipid disorders. These innovative treatments show promise in managing hypercholesterolemia and reducing cardiovascular risk.
Area of Science:
- Biochemistry and Molecular Biology
- Genetics and Genomics
- Pharmacology and Therapeutics
Background:
- Lipid disorders, including hypercholesterolemia, are significant risk factors for atherosclerotic cardiovascular disease.
- Advances in genetic medicine have opened new avenues for therapeutic interventions.
- RNA interference (RNAi) is a natural gene-silencing mechanism with therapeutic potential.
Purpose of the Study:
- To review current advances in RNA interference (RNAi) therapies, specifically small interfering RNA (siRNA) drugs.
- To critically assess the clinical positioning of these novel therapies.
- To explore the future role of RNAi in reshaping lipid management.
Main Methods:
- Review of current literature on RNAi therapies for lipid disorders.
- Analysis of clinical data for approved and investigational siRNA drugs.
- Assessment of the mechanism of action, efficacy, safety, and potential applications of RNAi therapies.
Main Results:
- RNAi therapies provide targeted and durable suppression of lipid-regulating proteins at the mRNA level.
- Inclisiran, an approved siRNA therapy, significantly reduces LDL-c levels with infrequent dosing.
- Investigational RNAi drugs target lipoprotein(a), apolipoprotein C-III, and angiopoietin-like protein 3 to address residual cardiovascular risk.
Conclusions:
- siRNA-based therapies represent a disruptive technology in lipidology, potentially transforming cardiovascular disease prevention and treatment.
- RNAi agents may become foundational in personalized lipid management, enabling long-acting and combination regimens.
- Ongoing trials are crucial to confirm cardiovascular benefits and safety, determining if RNAi becomes the future standard of care.
Related Concept Videos
01:20Lipid-Lowering Drugs: Statins and Miscellaneous Agents
1.6K
Hyperlipidemia, a medical condition often referred to as high cholesterol, is characterized by abnormally elevated levels of lipids in the bloodstream. When present in excess, these lipids, specifically cholesterol and triglycerides, can lead to serious health complications, often involving cardiovascular diseases. Illnesses like atherosclerosis, heart attacks, and pancreatitis have all been linked to untreated hyperlipidemia. This means controlling and regulating cholesterol and triglyceride...
1.6K
01:24Overview of Lipid Metabolism
6.2K
Lipid metabolism is a crucial process in the human body that involves the synthesis and degradation of lipids. This process is essential for energy production, cell membrane formation, and hormone production, among other functions.
Lipolysis: The Breakdown of Lipids:
Lipolysis is the process of breaking down lipids, particularly triglycerides, into glycerol and fatty acids. This process typically occurs in the adipose tissue and is triggered by various hormones, including glucagon and...
Lipolysis: The Breakdown of Lipids:
Lipolysis is the process of breaking down lipids, particularly triglycerides, into glycerol and fatty acids. This process typically occurs in the adipose tissue and is triggered by various hormones, including glucagon and...
6.2K
01:26Atherosclerosis III: Management
520
Management of atherosclerosis involves an integrated strategy encompassing pharmacological treatment, surgical interventions, lifestyle changes, and nutrition therapy to address the multifactorial nature of the disease.Pharmacological TherapyA cornerstone of atherosclerosis management is the use of pharmacological agents. Statins, such as atorvastatin, are pivotal in inhibiting HMG-CoA reductase, an enzyme that catalyzes an initial step in cholesterol synthesis in the liver. This reduction in...
520
01:31Transducer Mechanism: Nuclear Receptors
3.8K
Nuclear receptors, or NRs, are unique transcription factors that regulate gene transcription and affect the cellular pathways involved in reproduction, development, or metabolism. Their ability to be stimulated by small lipophilic ligands and control vital cellular processes makes them ideal drug targets. Nearly 10-15% of currently prescribed drugs target these receptors.
About 48 different soluble family members of nuclear receptors are identified that can be divided into two main classes:
About 48 different soluble family members of nuclear receptors are identified that can be divided into two main classes:
3.8K
01:28Assembly of the Lipid Bilayer in the ER
4.3K
Biological membranes are more than just a barrier separating cell cytoplasm from the outside environment. They are highly dynamic and help maintain the integrity and physiological stability of the cells as well as membrane-bound organelles. Membranes also play vital roles in cell-to-cell and intracellular communication.
A large chunk of any biological membrane is composed of phospholipids. These lipids have a heterogeneous distribution across different subcellular organelles and even between...
A large chunk of any biological membrane is composed of phospholipids. These lipids have a heterogeneous distribution across different subcellular organelles and even between...
4.3K
01:29Pharmacogenomics: Identification of New Drug Targets
53
Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
53