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Related Concept Videos

Nucleic acids02:43

Nucleic acids

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Nucleic acids are the most important macromolecules for the continuity of life. They carry the cell's genetic blueprint and carry instructions for its functioning.
DNA and RNA
The two main types of nucleic acids are deoxyribonucleic acid (DNA) and ribonucleic acid (RNA). DNA is the genetic material in all living organisms, ranging from single-celled bacteria to multicellular mammals. It is in the nucleus of eukaryotes and in the organelles, chloroplasts, and mitochondria. In prokaryotes,...
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Gene Therapy00:59

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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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Nucleic Acid Structure01:25

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The pentose sugar in DNA is deoxyribose, while in RNA the pentose sugar is ribose. The difference between the sugars is the presence of the hydroxyl group on the ribose's second carbon and a hydrogen on the deoxyribose's second carbon. The phosphate residue attaches to the hydroxyl group of the 5′ carbon of one sugar and the hydroxyl group of the 3′ carbon of the sugar of the next nucleotide, which forms  a 5′ to 3′ phosphodiester linkage.
DNA Structure
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Microorganisms in Medicine and Therapeutics01:29

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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.
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Related Experiment Video

Updated: Oct 21, 2025

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA
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Nucleic acid delivery for therapeutic applications.

Akash Gupta1, Jason L Andresen2, Rajith S Manan3

  • 1David H Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, 500 Main Street, Cambridge, MA 02139, USA.

Advanced Drug Delivery Reviews
|September 7, 2021
PubMed
Summary

Nucleic acid therapies, including siRNA, miRNA, ASO, and mRNA, offer new ways to treat diseases by targeting transcription and translation. This review highlights successful delivery strategies and clinical applications of these advanced therapies.

Keywords:
BiomaterialsCOVID-19CancerGenetic diseaseNucleic acid (oligonucleotide) deliveryTherapeuticsVaccines

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Last Updated: Oct 21, 2025

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Therapeutics

Background:

  • Medical advances now target diseases at the transcription and translation levels.
  • Nucleic acid-based therapies are emerging as a powerful treatment modality.

Purpose of the Study:

  • To review therapeutic applications of nucleic acids.
  • To highlight advancements in nucleic acid delivery technologies.
  • To discuss clinical progress and FDA-approved nucleic acid therapies.

Main Methods:

  • Review of recent literature on nucleic acid therapeutics.
  • Analysis of encapsulation and stabilization strategies for nucleic acid delivery.
  • Examination of clinical trial data and FDA approvals.

Main Results:

  • Various nucleic acids (siRNA, miRNA, ASO, mRNA) are used for gene knockdown or protein expression.
  • Novel delivery systems enhance intracellular uptake of nucleic acids.
  • Several nucleic acid therapies have reached late-stage clinical trials and gained FDA approval.

Conclusions:

  • Nucleic acid therapeutics represent a significant advancement in medicine.
  • Effective delivery systems are crucial for the success of these therapies.
  • The field shows great promise for treating a range of diseases.