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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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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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Polymalic Acid-based Nano Biopolymers for Targeting of Multiple Tumor Markers: An Opportunity for Personalized Medicine?
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Advanced Nanotechnology-Based Nucleic Acid Medicines.

Noriko Miyamoto1,2, Mina Sakuragi3, Yukio Kitade1,2,4

  • 1Department of Applied Chemistry, Faculty of Engineering, Aichi Institute of Technology, 1247 Yachigusa, Yakusa-cho, Toyota 470-0392, Japan.

Pharmaceutics
|November 27, 2024
PubMed
Summary

Nucleic acid medicines show promise but face delivery challenges. Nanotechnology offers solutions, with many nanotechnology-based nucleic acid medicines in clinical trials globally and in Japan.

Keywords:
ASOCpG-ODNDNAdrug delivery systemlipid nanoparticlesmRNAmiRNAnanotechnologynanotechnology-based medicinenucleic acid medicinepDNAsiRNA

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Area of Science:

  • Biotechnology and Pharmaceutical Sciences
  • Nanomedicine
  • Molecular Therapeutics

Background:

  • Nucleic acid medicines offer sequence-specific targeting of molecules.
  • 21 nucleic acid medicines are approved by the FDA.
  • Challenges remain in tissue targeting and intracellular delivery.

Purpose of the Study:

  • To review the latest advancements in nanotechnology for nucleic acid medicines.
  • To summarize the status of clinical trials for nanotechnology-based nucleic acid medicines in Japan.
  • To discuss future expectations for this therapeutic modality.

Main Methods:

  • Literature review of recent research in nanotechnology applied to nucleic acid medicines.
  • Analysis of clinical trial data for nanotechnology-based nucleic acid medicines, with a focus on Japan.
  • Synthesis of current trends and future prospects.

Main Results:

  • Over 40 nanotechnology-based nucleic acid medicines have entered clinical trials outside Japan, with 15 currently active.
  • Three Phase I clinical trials involving nanotechnology-based nucleic acid medicines are ongoing in Japan.
  • Nanotechnology is actively being employed to overcome delivery and targeting challenges.

Conclusions:

  • Nanotechnology is crucial for advancing nucleic acid medicine development.
  • Clinical trials in Japan are progressing, with results anticipated.
  • Further evolution of nanotechnology in nucleic acid medicines is expected.