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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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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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Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

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Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
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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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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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Gene Therapy00:59

Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Related Experiment Video

Updated: Aug 28, 2025

Development of a 68Gallium-Labeled D-Peptide PET Tracer for Imaging Programmed Death-Ligand 1 Expression
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RNA therapeutics: updates and future potential.

Caroline Zhang1, Biliang Zhang2,3

  • 1Guangzhou RiboBio Co., Ltd, Guangzhou, 510663, China.

Science China. Life Sciences
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This summary is machine-generated.

RNA therapeutics are rapidly advancing, offering new treatments for previously undruggable diseases. This review covers five key RNA therapy types and their clinical progress.

Keywords:
RNARNA therapeuticsRNAimRNA vaccine

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • Advancements in RNA production, modification, and delivery have spurred the growth of RNA-based therapeutics.
  • A deeper understanding of RNA biology underpins the expansion of this therapeutic modality.

Purpose of the Study:

  • To review the general concepts of five major classes of RNA-based therapeutics.
  • To provide an overview of approved and clinically evaluated RNA-based therapies.
  • To discuss the challenges and future potential of RNA therapeutics.

Main Methods:

  • Literature review of RNA-based therapeutic classes.
  • Analysis of regulatory approvals and clinical trial data.
  • Discussion of technological challenges and opportunities.

Main Results:

  • Five classes of RNA therapeutics are detailed: RNA interference, antisense oligonucleotides, small activating RNA, circular RNA, and messenger RNA.
  • Several RNA-based therapies have achieved regulatory approval or are in clinical trials.
  • Positive clinical results highlight the potential of RNA drugs.

Conclusions:

  • RNA-based therapeutics represent a promising, disruptive technology with the potential to address unmet medical needs.
  • These therapies can target previously undruggable targets, offering new hope for various diseases.
  • Continued research and development are crucial to overcome challenges and realize the full clinical potential of RNA therapeutics.