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Related Concept Videos

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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 ATP-dependent...
Experimental RNAi02:15

Experimental RNAi

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...
RNA Interference01:23

RNA Interference

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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Related Experiment Video

Updated: May 18, 2026

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes
10:33

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Published on: July 23, 2016

Lipid-based vectors for siRNA delivery.

Shubiao Zhang1, Defu Zhi, Leaf Huang

  • 1Division of Molecular Pharmaceutics, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.

Journal of Drug Targeting
|September 22, 2012
PubMed
Summary
This summary is machine-generated.

Developing effective siRNA delivery systems is crucial for therapeutics. Lipid-based vectors show promise, with novel lipidoids and complex structures like LPDs offering potential solutions to current delivery challenges.

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Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy
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Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy

Published on: June 21, 2013

Porous Silicon Microparticles for Delivery of siRNA Therapeutics
08:31

Porous Silicon Microparticles for Delivery of siRNA Therapeutics

Published on: January 15, 2015

Related Experiment Videos

Last Updated: May 18, 2026

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes
10:33

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Published on: July 23, 2016

Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy
15:55

Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy

Published on: June 21, 2013

Porous Silicon Microparticles for Delivery of siRNA Therapeutics
08:31

Porous Silicon Microparticles for Delivery of siRNA Therapeutics

Published on: January 15, 2015

Area of Science:

  • Biotechnology
  • Nanomedicine
  • Molecular Biology

Background:

  • RNA interference (RNAi) therapeutics, particularly small interfering RNA (siRNA), are advancing rapidly with ongoing clinical trials.
  • Efficient delivery of siRNA into target cells, tissues, and organs remains a significant hurdle for therapeutic applications.
  • Lipid-based non-viral vectors are attractive due to their structural similarity to biological membranes, but traditional liposomes present limitations.

Purpose of the Study:

  • To explore advanced lipid-based delivery systems for siRNA therapeutics.
  • To evaluate novel materials like lipidoids and complex self-assembly structures for improved siRNA delivery.
  • To address the challenges associated with current siRNA vector technologies.

Main Methods:

  • Investigated lipidoids as a novel class of siRNA delivery materials, noting their ease of synthesis and potent silencing activity.
  • Examined pH-responsive lipids, including amine-based structures, for their potential in targeted siRNA delivery.
  • Studied complex self-assembly structures, such as Lipid-Protide-Dendrimer (LPD) and Lipid-Cationic Polymer (LCP) complexes, for their delivery capabilities.

Main Results:

  • Lipidoids demonstrate excellent siRNA silencing activity, though their toxicological profiles require further investigation.
  • pH-responsive lipids have garnered interest, with some amine-based lipids showing promise despite lacking novel chemical structures.
  • LPD and LCP structures exhibit controlled particle morphology and size, facilitating effective siRNA delivery both in vitro and in vivo.

Conclusions:

  • Novel lipidoids and advanced lipid-based formulations like LPDs and LCPs represent promising strategies to overcome current siRNA delivery obstacles.
  • Further research into the safety and efficacy of these novel delivery systems is warranted for clinical translation.
  • Optimizing lipid-based vector design is key to unlocking the full therapeutic potential of siRNA technology.