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Related Concept Videos

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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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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siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
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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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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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Tumor Immunotherapy01:27

Tumor Immunotherapy

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Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.
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Related Experiment Video

Updated: Oct 16, 2025

Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism
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Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism

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Oligonucleotides-Based Therapeutics.

Bernard Lebleu1

  • 1Laboratory of Pathogen Host Interactions, Université Montpellier, UMR 5235 CNRS, 34000 Montpellier, France.

Biomedicines
|October 23, 2021
PubMed
Summary

Nucleic-acid-based strategies are revolutionizing medicine by enabling precise gene expression regulation. These innovative approaches are paving the way for a new era of targeted therapies.

Area of Science:

  • Molecular Biology
  • Genetics
  • Pharmacology

Background:

  • Nucleic acids offer precise tools for modulating gene expression.
  • Emerging nucleic-acid-based therapies represent a novel class of medicines.

Discussion:

  • Exploring the mechanisms of action for various nucleic-acid-based strategies.
  • Addressing challenges in delivery and stability for therapeutic applications.
  • Highlighting the potential to target previously undruggable genetic disorders.

Key Insights:

  • Gene expression regulation via nucleic acids is a rapidly advancing field.
  • These strategies offer unprecedented specificity in therapeutic interventions.
  • The development of nucleic-acid-based medicines is transforming personalized medicine.

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Nucleoside Triphosphates - From Synthesis to Biochemical Characterization
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Nucleoside Triphosphates - From Synthesis to Biochemical Characterization

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Related Experiment Videos

Last Updated: Oct 16, 2025

Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism
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Protocol for the Solid-phase Synthesis of Oligomers of RNA Containing a 2'-O-thiophenylmethyl Modification and Characterization via Circular Dichroism

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Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

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Nucleoside Triphosphates - From Synthesis to Biochemical Characterization
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Nucleoside Triphosphates - From Synthesis to Biochemical Characterization

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Outlook:

  • Future research directions in optimizing nucleic-acid-based therapies.
  • Expanding the therapeutic landscape for genetic diseases.
  • Integration of artificial intelligence in designing novel nucleic-acid drugs.