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Related Concept Videos

Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

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...
Lipid-Lowering Drugs: Statins and Miscellaneous Agents01:20

Lipid-Lowering Drugs: Statins and Miscellaneous Agents

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...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Targets for Drug Action: Overview01:26

Targets for Drug Action: Overview

Drugs target macromolecules to modify ongoing cellular processes. Primary drug targets include receptors, ion channels, transporters, and enzymes.
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand, get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either mimicking the action of endogenous ligands or blocking the receptor activity to bring about a modified response. Nearly 35% of approved drugs target the G...
Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase01:11

Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase

Genetic polymorphisms in drug targets have emerged as critical determinants of interindividual variability in drug response and toxicity. Pharmacogenomic investigations increasingly focus on identifying these variations to personalize and optimize therapeutic interventions. A drug target may be a receptor, enzyme, or signaling protein involved in pharmacologic responses or disease-related pathways. While early pharmacogenetic studies focused primarily on drug metabolism, current research...

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Related Experiment Video

Updated: May 22, 2026

LDL Cholesterol Uptake Assay Using Live Cell Imaging Analysis with Cell Health Monitoring
08:45

LDL Cholesterol Uptake Assay Using Live Cell Imaging Analysis with Cell Health Monitoring

Published on: November 17, 2018

RNA-Targeted Therapeutics for Lipid Metabolic Disorders: From Bench to Bedside.

Iqra Ali1,2, Juhui Qiu1, Wai San Cheang3

  • 1Key Laboratory for Biorheological Science and Technology of Ministry of Education, State and Local Joint Engineering Laboratory for Vascular Implants, College of Bioengineering, Chongqing University, Chongqing 400030, China.

Research (Washington, D.C.)
|May 21, 2026
PubMed
Summary

RNA therapeutics offer a novel approach to lower lipids and reduce cardiovascular disease risk. Targeting specific genes with RNA-based drugs, like antisense oligonucleotides, provides durable lipid reduction, improving patient outcomes.

Related Experiment Videos

Last Updated: May 22, 2026

LDL Cholesterol Uptake Assay Using Live Cell Imaging Analysis with Cell Health Monitoring
08:45

LDL Cholesterol Uptake Assay Using Live Cell Imaging Analysis with Cell Health Monitoring

Published on: November 17, 2018

Area of Science:

  • Cardiovascular Medicine
  • Genetics
  • Pharmacology

Background:

  • Atherosclerotic cardiovascular disease, a leading cause of death, is driven by lipid accumulation.
  • Current lipid-lowering drugs leave substantial residual risk, despite evidence supporting lower LDL cholesterol goals.
  • The liver's central role in lipid metabolism makes it a key target for therapeutic intervention.

Purpose of the Study:

  • To provide a state-of-the-art overview of RNA-targeted therapeutics for hyperlipidemia.
  • To discuss the potential of RNA-based therapies in managing cardiovascular disease.
  • To explore future research directions in RNA-targeted lipid modulation.

Main Methods:

  • Review of recent clinical evidence and guidelines (e.g., 2026 ACC/AHA dyslipidemia guideline).
  • Discussion of RNA therapeutics including antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) conjugated with N-acetylgalactosamine (GalNAc) for hepatocyte-specific delivery.
  • Exploration of novel approaches like in vivo base editing using mRNA lipid nanoparticles.

Main Results:

  • RNA therapeutics, targeting genes like PCSK9, APOC3, Lp(a), and ANGPTL3, offer durable silencing of lipid targets.
  • Hepatocyte-specific delivery via GalNAc enhances efficacy of RNA-based drugs.
  • Emerging technologies like in vivo base editing show potential for permanent genetic correction.

Conclusions:

  • RNA-targeted therapeutics represent a transformative approach to hyperlipidemia, achieving very low LDL cholesterol levels.
  • Future advancements in extrahepatic delivery and combination regimens may lead to disease-modifying interventions.
  • Targeting PCSK9, APOC3, Lp(a), and ANGPTL3 is clinically emphasized for improved cardiovascular outcomes.