Related Experiment Video
Updated: Feb 20, 2026

05:28
A Positioning Device for the Placement of Mice During Intranasal siRNA Delivery to the Central Nervous System
Published on: August 15, 2019
25.5K
Taking charge of siRNA delivery
Anastasia Khvorova1, Maire F Osborn1, Matthew R Hassler1
1RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.
Nature Biotechnology
|December 10, 2014
Summary
This study presents a novel method for delivering small interfering RNA (siRNA) into cells. The technique involves temporarily neutralizing the siRNA
Area of Science:
- Biochemistry
- Molecular Biology
- Cell Biology
Background:
- Small interfering RNA (siRNA) is a powerful tool for gene silencing.
- Efficient delivery of siRNA into target cells remains a significant challenge in molecular biology.
- The negatively charged phosphate backbone of siRNA hinders cellular uptake.
Purpose of the Study:
- To develop a method for effective and reversible siRNA delivery into cells.
- To overcome the charge-related barriers for intracellular siRNA transport.
Main Methods:
- Utilizing a reversible charge neutralization strategy for siRNA.
- Investigating the mechanism of siRNA delivery via charge modification.
Main Results:
- Successfully demonstrated reversible charge neutralization of siRNA.
- Achieved efficient delivery of siRNA into cells using this approach.
Conclusions:
- Reversible charge neutralization is a viable strategy for enhancing siRNA delivery.
- This method offers a promising avenue for therapeutic applications of RNA interference.
Related Concept Videos
Translational Regulation
696
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,...
696
siRNA - Small Interfering RNAs
18.8K
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...
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...
18.8K
RNA Interference
28.2K
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...
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...
28.2K
Tail-anchoring of Proteins in the ER Membrane
3.9K
Tail-anchored, or TA, proteins are estimated to make up to 3-5% of membrane proteins found in the eukaryotic cell. Such proteins have a single transmembrane domain located approximately 30 amino acid residues upstream from the C-terminal end. As a result, the signal recognition particle (SRP) cannot guide a TA protein to the ER membrane for cotranslational insertion. Hence, they are integrated into the ER membrane post-translationally using their C-terminal end as the anchor. TA proteins...
3.9K
Directing Proteins to the Rough Endoplasmic Reticulum
18.0K
The organelle-specific signaling sequences direct proteins synthesized in the cytosol to their final destination like ER, mitochondria, peroxisomes, etc. Some of the proteins directed to ER are then trafficked via vesicles to other organelles within the cell or the extracellular environment through the Golgi complex. For example, the rough ER synthesizes soluble proteins for transportation to the lysosomes or secretion out of the cell. It can also synthesize transmembrane proteins that can...
18.0K
Regulation of Nuclear Protein Sorting
3.4K
Nuclear protein sorting regulates nucleus composition and gene expression, crucial for determining the fate of a eukaryotic cell. Hence, the entry and exit of molecules across the nuclear envelope is a tightly controlled process. Nuclear protein sorting can be inhibited by one of the following ways: 1) masking cargo signal sequences, 2) modifying the nuclear receptor's affinity for cargo, 3) controlling the nuclear pore size, 4) retaining the cargo during its transit to the cytosol or the...
3.4K

