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Related Concept Videos

Experimental RNAi02:15

Experimental RNAi

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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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Types of RNA01:20

Types of RNA

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Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in regulating gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA Performs Diverse...
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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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lncRNA - Long Non-coding RNAs02:39

lncRNA - Long Non-coding RNAs

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In humans, more than 80% of the genome gets transcribed. However, only around 2% of the genome codes for proteins. The remaining part produces non-coding RNAs which includes ribosomal RNAs, transfer RNAs, telomerase RNAs, and regulatory RNAs, among other types. A large number of regulatory non-coding RNAs have been classified into two groups depending upon their length – small non-coding RNAs, such as microRNA, which are less than 200 nucleotides in length, and long non-coding RNA...
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Translational Regulation01:29

Translational Regulation

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Translational regulation in prokaryotes ensures efficient protein synthesis by controlling ribosome access to mRNA. This regulation is mediated by secondary RNA structures, including translational riboswitches, RNA thermometers, and small RNAs (sRNAs), which respond to intracellular and environmental signals to modulate gene expression.Translational RiboswitchesRiboswitches in the leader region of mRNAs can regulate translation by altering the accessibility of the Shine-Dalgarno (SD) sequence,...
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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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Emerging Approaches for Enabling RNAi Therapeutics.

Argha M Mallick1, Archana Tripathi1, Sukumar Mishra1

  • 1Department of Biological Sciences, Indian Institute of Science Education and Research Kolkata, Mohanpur, 741246, India.

Chemistry, an Asian Journal
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Summary

RNA interference (RNAi) therapeutics, using small interfering RNA (siRNA), require efficient delivery vehicles. This review explores designing principles and examples of these vehicles for genetic diseases and cancer therapy.

Keywords:
drug deliveryendosomal escape, lipidsgene silencingpeptides.polymerssiRNA

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • RNA interference (RNAi) is an evolutionary mechanism for defense against foreign genetic material.
  • Small interfering RNA (siRNA) offers therapeutic potential for gene silencing.
  • Effective delivery of siRNA is crucial for its therapeutic efficacy, requiring vehicles to overcome physiological barriers and reach the RISC complex.

Purpose of the Study:

  • To review the design principles and examples of delivery vehicles for RNAi therapeutics.
  • To discuss the applications of RNAi therapeutics in genetic diseases, epigenetic modifications, and cancer therapy.
  • To explore strategies and opportunities for developing clinically translatable RNAi delivery vehicles.

Main Methods:

  • Literature review of existing research on RNAi delivery vehicles.
  • Analysis of designing principles for various classes of delivery systems.
  • Discussion of therapeutic applications and future directions in RNAi delivery.

Main Results:

  • Various classes of delivery vehicles have demonstrated efficiency in RNAi therapeutics.
  • RNAi holds promise for treating genetic disorders, epigenetic modifications, and enabling personalized cancer therapy.
  • Significant progress has been made in developing strategies for efficient siRNA delivery.

Conclusions:

  • Efficient delivery vehicles are essential for realizing the full therapeutic potential of siRNA.
  • Continued research into delivery systems is critical for advancing RNAi-based treatments for various diseases.
  • Translating these advancements into clinical practice requires innovative strategies and further development.