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!
Seeing on the Quick: New Insights into Active Vision in the Brain
15.08.2018 | Eberhard Karls Universität Tübingen
New Approach to Treating Chronic Itch
15.08.2018 | Universität Zürich
Scientists at the University of California, Los Angeles present new research on a curious cosmic phenomenon known as "whistlers" -- very low frequency packets...
Scientists develop first tool to use machine learning methods to compute flow around interactively designable 3D objects. Tool will be presented at this year’s prestigious SIGGRAPH conference.
When engineers or designers want to test the aerodynamic properties of the newly designed shape of a car, airplane, or other object, they would normally model...
Researchers from TU Graz and their industry partners have unveiled a world first: the prototype of a robot-controlled, high-speed combined charging system (CCS) for electric vehicles that enables series charging of cars in various parking positions.
Global demand for electric vehicles is forecast to rise sharply: by 2025, the number of new vehicle registrations is expected to reach 25 million per year....
Proteins must be folded correctly to fulfill their molecular functions in cells. Molecular assistants called chaperones help proteins exploit their inbuilt folding potential and reach the correct three-dimensional structure. Researchers at the Max Planck Institute of Biochemistry (MPIB) have demonstrated that actin, the most abundant protein in higher developed cells, does not have the inbuilt potential to fold and instead requires special assistance to fold into its active state. The chaperone TRiC uses a previously undescribed mechanism to perform actin folding. The study was recently published in the journal Cell.
Actin is the most abundant protein in highly developed cells and has diverse functions in processes like cell stabilization, cell division and muscle...
Scientists have discovered that the electrical resistance of a copper-oxide compound depends on the magnetic field in a very unusual way -- a finding that could help direct the search for materials that can perfectly conduct electricity at room temperatur
What happens when really powerful magnets--capable of producing magnetic fields nearly two million times stronger than Earth's--are applied to materials that...
08.08.2018 | Event News
27.07.2018 | Event News
25.07.2018 | Event News
15.08.2018 | Physics and Astronomy
15.08.2018 | Earth Sciences
15.08.2018 | Physics and Astronomy