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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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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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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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Progress on RNA-based therapeutics for genetic diseases.

Ting Luo1, Chunxiao Huo2, Tianhua Zhou3,4,5

  • 1Department of Cell Biology, Zhejiang University School of Medicine, Hangzhou 310058, China. tingluo1117@163.com.

Zhejiang Da Xue Xue Bao. Yi Xue Ban = Journal of Zhejiang University. Medical Sciences
|August 29, 2023
PubMed
Summary

RNA therapeutics offer new ways to treat genetic diseases by targeting specific genes. Research focuses on improving RNA drug efficacy and delivery through chemical modifications and advanced systems.

Keywords:
Antisense oligonucleotidesGenetic diseaseMessenger RNA drugsRNA aptamersRNA, drugRNA, medicineReviewSmall interfering RNA

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • RNA therapeutics offer targeted gene expression modulation for genetic disease treatment.
  • Current RNA-based drugs include oligonucleotide and mRNA types, with varying regulatory approvals.

Purpose of the Study:

  • To provide an overview of RNA drug molecular mechanisms.
  • To discuss advancements in chemical modifications and delivery systems for RNA therapeutics.

Main Methods:

  • Review of molecular mechanisms of eleven RNA drugs.
  • Analysis of research progress in chemical modifications (e.g., pseudouridine).
  • Examination of delivery systems (e.g., lipid nanoparticles, extracellular vesicles).

Main Results:

  • Eleven RNA drugs targeting specific genes are detailed.
  • Chemical modifications enhance RNA drug efficacy and reduce immunogenicity.
  • Advanced delivery systems are crucial for RNA drug stability, specificity, and safety.

Conclusions:

  • RNA therapeutics hold significant promise for treating genetic diseases.
  • Ongoing research in modifications and delivery systems is vital for clinical translation.