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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

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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.
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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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Site-Targeted Drug Delivery Systems: Polymeric Carriers01:24

Site-Targeted Drug Delivery Systems: Polymeric Carriers

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Polymeric carriers enhance targeted drug delivery by increasing efficacy while minimizing off-target effects. These carriers comprise a biodegradable polymeric backbone integrated with functional elements that enable targeting, improve physicochemical properties, and regulate drug release.Targeting MechanismsThe targeting ability of polymeric carriers is mediated by a homing device, which is a molecular recognition component designed to selectively bind to specific tissues or cells. Monoclonal...
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Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA
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Lipid-Based Nanocarriers for RNA Delivery.

Hui Yi Xue, Pengbo Guo, Wu-Cheng Wen

  • 1School of Pharmacy, Temple University, 3307 North Broad Street, Philadelphia, Pennsylvania, US 19140. ho-lun.wong@temple.edu.

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Lipid-based nanocarriers show promise for delivering RNA interference (RNAi) therapeutics, including small-interfering RNA (siRNA), to treat various diseases. This review discusses challenges and strategies for optimizing these delivery systems for clinical success.

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Area of Science:

  • Biomedical Engineering
  • Nanotechnology
  • Pharmacology

Background:

  • RNA-interference (RNAi) agents like siRNA and miRNA offer therapeutic potential for diseases linked to aberrant gene expression.
  • Clinical trials of RNAi therapeutics have faced challenges, highlighting the need for effective delivery systems.
  • Lipid-based nanocarriers are widely used for RNA delivery due to their biocompatibility and biodegradability.

Purpose of the Study:

  • To review major lipid-based nanocarrier subclasses for RNA delivery.
  • To discuss challenges in developing these nanocarriers, such as cationic lipid toxicity and PEGylated lipid issues.
  • To explore strategies for overcoming these obstacles in RNAi therapy.

Main Methods:

  • Review of literature on lipid-based nanocarriers for RNA delivery.
  • Analysis of key features of liposomes, lipid nanoparticles, and lipid nanoemulsions.
  • Discussion of challenges and solutions in nanocarrier development.

Main Results:

  • Lipid-based nanocarriers (liposomes, lipid nanoparticles, lipid nanoemulsions) are crucial for RNA delivery.
  • Development challenges include toxicity of cationic lipids and problems with PEGylated lipids.
  • Strategies exist to mitigate these issues, improving RNA delivery system design.

Conclusions:

  • Understanding the advantages and disadvantages of lipid-based RNA delivery systems is vital.
  • Overcoming current obstacles can enhance the clinical translation of RNAi therapeutics.
  • This knowledge aids pharmaceutical scientists, researchers, and clinicians in advancing RNAi therapy.