Scientists have been exploring RNA interference (RNAi) as a gene therapy technique to silence genes that are improperly produced. The "RNAi" approach requires the delivery into the cell of short pieces of the genetic material Ribonucleic Acid (RNA).
These synthetic short RNA "oligos" can then pair with specific sites in the cell's own RNA, targeting the genetic messages for destruction and turning off expression of the corresponding genes. However, the widespread clinical use of this genetic therapy relies upon technical improvements, including new delivery vehicles such as the one Kent Kirshenbaum, an assistant professor in NYU's Department of Chemistry, and colleagues present in their work.
The NYU researchers use a modular linear molecule to deliver therapeutic RNA into cells. The molecule has a positively charged site that forms favorable stabilizing interactions with the negatively charged RNA, and a fatty component that interacts with cell membranes. The molecules and RNA form complexes, which protect the RNA from being degraded and deliver it to cells. As a result, the targeted deleterious genes are silenced.
Their research concentrates on making the transition from the lab into real-life smoother. Their linear molecule can be used to deliver small therapeutic RNAs into cell types that are much more representative of cellular targets that investigators are likely to encounter in clinical situations.
Kirshenbaum and his coworkers are now focused on understanding the physical chemical characteristics that give enhanced activity to their molecule, and then use the knowledge to generate a set of more sophisticated delivery reagents for siRNA.
"Our goal is to develop a platform that would allow us to create a library that could be used in different settings or for delivery to different cell types," he told Chemical and Engineering News.
James Devitt | EurekAlert!
Ion treatments for cardiac arrhythmia — Non-invasive alternative to catheter-based surgery
20.01.2017 | GSI Helmholtzzentrum für Schwerionenforschung GmbH
Seeking structure with metagenome sequences
20.01.2017 | DOE/Joint Genome Institute
An important step towards a completely new experimental access to quantum physics has been made at University of Konstanz. The team of scientists headed by...
Yersiniae cause severe intestinal infections. Studies using Yersinia pseudotuberculosis as a model organism aim to elucidate the infection mechanisms of these...
Researchers from the University of Hamburg in Germany, in collaboration with colleagues from the University of Aarhus in Denmark, have synthesized a new superconducting material by growing a few layers of an antiferromagnetic transition-metal chalcogenide on a bismuth-based topological insulator, both being non-superconducting materials.
While superconductivity and magnetism are generally believed to be mutually exclusive, surprisingly, in this new material, superconducting correlations...
Laser-driving of semimetals allows creating novel quasiparticle states within condensed matter systems and switching between different states on ultrafast time scales
Studying properties of fundamental particles in condensed matter systems is a promising approach to quantum field theory. Quasiparticles offer the opportunity...
Among the general public, solar thermal energy is currently associated with dark blue, rectangular collectors on building roofs. Technologies are needed for aesthetically high quality architecture which offer the architect more room for manoeuvre when it comes to low- and plus-energy buildings. With the “ArKol” project, researchers at Fraunhofer ISE together with partners are currently developing two façade collectors for solar thermal energy generation, which permit a high degree of design flexibility: a strip collector for opaque façade sections and a solar thermal blind for transparent sections. The current state of the two developments will be presented at the BAU 2017 trade fair.
As part of the “ArKol – development of architecturally highly integrated façade collectors with heat pipes” project, Fraunhofer ISE together with its partners...
19.01.2017 | Event News
10.01.2017 | Event News
09.01.2017 | Event News
20.01.2017 | Awards Funding
20.01.2017 | Materials Sciences
20.01.2017 | Life Sciences