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Related Concept Videos

RNA Interference01:23

RNA Interference

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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
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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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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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Types of RNA01:23

Types of RNA

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Overview
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 the regulation of 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...
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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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Small interfering RNAs (siRNA)02:30

Small interfering RNAs (siRNA)

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Emerging Trends in Micro- and Nanoscale Technologies in Medicine: From Basic Discoveries to Translation.

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Next-Generation Lipids in RNA Interference Therapeutics.

Stephanie Rietwyk1, Dan Peer1

  • 1Laboratory of Precision NanoMedicine, Department of Cell Research & Immunology, George S. Wise Faculty of Life Sciences, ‡Department of Materials Sciences and Engineering, Iby and Aladar Fleischman Faculty of Engineering, §Center for Nanoscience and Nanotechnology, and ∥Cancer Biology Research Center, Tel Aviv University , Tel Aviv 69978, Israel.

ACS Nano
|July 21, 2017
PubMed
Summary

Lipid nanoparticles (LNPs) are crucial for delivering RNA therapeutics but face challenges beyond the liver. Next-generation ionizable lipids improve safety and endosomal escape for better therapeutic delivery.

Keywords:
RNA interferenceRNAi therapeuticscationic lipidsdeliverygene silencingionizable amino lipidslipid nanoparticleslipidoidssiRNAtoxicity

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Area of Science:

  • Biotechnology
  • Gene Therapy
  • Drug Delivery Systems

Background:

  • RNA therapeutics show promise for incurable diseases but require effective delivery systems.
  • Lipid nanoparticles (LNPs) are leading nonviral vectors for gene delivery, yet face limitations.
  • Ineffective delivery beyond the liver and toxicity concerns hinder clinical translation.

Purpose of the Study:

  • To review the limitations of current lipid nanoparticle (LNP) delivery systems for RNA therapeutics.
  • To discuss the challenges of LNP delivery beyond the liver and associated toxicities.
  • To explore next-generation ionizable cationic lipids as improved delivery vehicles.

Main Methods:

  • Critical literature review of LNP technology for RNA delivery.
  • Analysis of toxicity issues with permanently charged cationic lipids.
  • Evaluation of ionizable cationic lipids for enhanced endosomal escape and reduced toxicity.

Main Results:

  • Ineffective delivery beyond the liver is a significant barrier for current LNPs.
  • Permanently charged cationic lipids present toxicity and immunogenicity concerns.
  • Ionizable cationic lipids demonstrate reduced toxicity and improved endosomal escape for cytosolic delivery.

Conclusions:

  • Next-generation ionizable lipids represent a promising advancement in LNP technology for RNA therapeutics.
  • Overcoming delivery barriers and toxicity is essential for broader clinical translation of LNP-based RNA therapies.
  • Further research and clinical trials are needed to optimize LNP formulations and expand their therapeutic applications.