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Related Concept Videos

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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Nucleic Acid Structure01:25

Nucleic Acid Structure

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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
DNA...
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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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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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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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What is Genetic Engineering?00:49

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Related Experiment Video

Updated: Dec 10, 2025

Synthesis of Stimuli-responsive Nanogels using Aqueous One-step Crosslinking and Co-nanopolymerization
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Gene Therapy Based on Nucleic Acid Nanostructure.

Xiaohui Wu1,2, Tiantian Wu1,2, Jianbing Liu1,2

  • 1CAS Key Laboratory of Nanosystem and Hierarchical Fabrication, CAS Center for Excellence in Nanoscience, National Center for NanoScience and Technology, Beijing, 100190, China.

Advanced Healthcare Materials
|September 1, 2020
PubMed
Summary

Nucleic acid nanostructures offer programmable and biocompatible platforms for advanced biomedical applications. This review highlights their use in gene therapy delivery systems, addressing challenges and future opportunities.

Keywords:
drug deliverygene therapynucleic acid drugsnucleic acid nanostructures

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Area of Science:

  • Biotechnology
  • Nanotechnology
  • Molecular Biology

Background:

  • Nucleic acids are utilized to create programmable, addressable, and biocompatible nanostructures.
  • These nanostructures have broad biomedical applications including bioimaging, biosensing, and drug delivery.

Purpose of the Study:

  • To review recent advancements in multifunctional nucleic acid nanocarriers for gene therapy.
  • To discuss the potential of nucleic acid nanostructures for delivering gene-related drugs.

Main Methods:

  • Review of literature on nucleic acid nanocarrier design and applications.
  • Focus on gene therapy strategies including antisense, RNA interference, gene editing, and gene expression.

Main Results:

  • Nucleic acid nanostructures show promise as carriers for gene therapy applications.
  • Multifunctional nanocarriers are being designed for targeted gene delivery.

Conclusions:

  • Nucleic acid nanotechnology presents significant opportunities for gene therapy.
  • Challenges remain in optimizing nanocarrier design and delivery for clinical translation.