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Related Concept Videos

Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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Targets for Drug Action: Overview01:26

Targets for Drug Action: Overview

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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...
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Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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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...
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Transducer Mechanism: Enzyme-Linked Receptors01:27

Transducer Mechanism: Enzyme-Linked Receptors

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Enzyme-linked receptors are cell-surface receptors acting as an enzyme or associating with an enzyme intracellularly. They make excellent drug targets. Drugs can bind to the extracellular ligand-binding domain or directly affect their enzymatic domain and alter their activity.
Major types that are helpful drug targets include:
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Transducer Mechanism: Nuclear Receptors01:31

Transducer Mechanism: Nuclear Receptors

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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:
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Principles of Drug Action01:24

Principles of Drug Action

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Drugs are chemical substances that modify biological responses by interacting with macromolecular targets such as receptors, ion channels, transporters, and enzymes. Pharmacodynamics describes the course of action of drugs leading to the physiological effect at a specific site in the body.
Drugs can be agonists or antagonists. Like the endogenous ligands, agonists always bind and activate the target to produce a cellular response. Agonist binding induces a conformational change which in turn...
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Related Experiment Video

Updated: Jan 10, 2026

Synthesis of Stimuli-responsive Nanogels using Aqueous One-step Crosslinking and Co-nanopolymerization
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Synthesis of Stimuli-responsive Nanogels using Aqueous One-step Crosslinking and Co-nanopolymerization

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RNA-based drugs: current, imminent and possible therapeutic applications.

Sebastiano A Torrisi1, Federica Geraci2, Lidia Diolosà3

  • 1Department of Biomedical and Biotechnological Sciences, University of Catania, Catania, Italy; Oasi Research Institute-IRCCS, Unit of Translational Neuropharmacology and Translational Neurosciences, Troina, Italy.

Pharmacology & Therapeutics
|November 20, 2025
PubMed
Summary

Messenger RNA (mRNA) vaccines have saved millions of lives, paving the way for novel RNA-based therapeutics. These versatile drugs offer cost-effective treatments for previously undruggable targets across diverse diseases.

Keywords:
ASOAptamerCancerTherapymRNA vaccinemiRNAsiRNA

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Area of Science:

  • Pharmacology
  • Biotechnology
  • Drug Discovery

Background:

  • Messenger RNA (mRNA) vaccines have demonstrated significant success in combating the COVID-19 pandemic, saving millions of lives.
  • The development of RNA-based therapeutics represents a paradigm shift in medicine, offering promising applications beyond infectious diseases.
  • RNA-based drugs are characterized by ease of design, manufacturing, and potential cost-effectiveness.

Purpose of the Study:

  • To provide a comprehensive pharmacological analysis of the current RNA therapeutics landscape.
  • To review approved RNA-based drugs and key investigational candidates.
  • To highlight clinical progress, pharmacological mechanisms, and challenges in RNA drug development.

Main Methods:

  • Literature review of approved and investigational RNA-based drugs.
  • Analysis of pharmacological mechanisms, drug delivery challenges, tolerability, and clinical outcomes.
  • Exploration of RNA-based drug repurposing strategies.

Main Results:

  • RNA-based drugs can target previously undruggable pharmacological targets, enabling personalized treatments for difficult-to-treat diseases.
  • These therapeutics show versatility across various diseases, including cancer, neurodegenerative, cardiovascular, metabolic, rare genetic, and infectious diseases.
  • Drug repurposing leveraging shared pathophysiological mechanisms can accelerate clinical impact.

Conclusions:

  • RNA-based therapeutics represent a significant advancement in medicine with broad therapeutic potential.
  • Addressing challenges in drug delivery and optimizing tolerability are crucial for clinical success.
  • The versatility and potential for repurposing of RNA-based drugs offer a promising future for personalized medicine.