Adeno-associated virus (AAV) vectors, in particular serotype AAV9, are the most potent and promising vector types used for gene transfer into the heart. However AAV vectors also transduce non-target organs such as liver or skeletal muscle. Reduction of AAV-mediated transgene expression in non-target organs is an important aim to reduce or avoid unwanted side effects in gene therapy. The use of heart-specific promoters or the use of non-target organ specific microRNAs (miRNA) and its specific target site are approaches to control transgene transcription. E.g. the use of a miR-122 target site in the 3´UTR of an AAV9 vector leads to silencing of the transgene expression in the non-target organ liver (miR-122 is a liver-specific microRNA) (Geisler et al. 2011). <br><br> <strong>Technology</strong><br> We offer a mutated miR-206 target site (TS) as a control element for the si-lencing of unwanted transgene expression in the non-target organ skeletal muscle while allowing transgene expression in the target organ (e.g. heart). miR-206 is highly expressed in skeletal muscle and virtually absent in the heart. Unexpectedly, the expression of wild-type miR-206TS in the AAV9 transgene vector indeed exhibited reduced expression of the transgene in skeletal muscle however transgene expression was also silenced in the heart. This was due to the presence of miR-1 in the heart which shows 86% homology to miR-206. Thus miR-1 in the heart also binds to miR-206TS in the AAV9 vector which results in silencing of transgene expression in the heart. The use of specifically mutated miR-206TS overcomes this problem: miR-206 binds furthermore and even better to the mutated miR-206TS whereas miR-1 cannot bind the mutated miR-206TS. Hence, transgene expression is repressed in the skeletal muscle whereas it remains high in the heart.<br><br>
email@example.com | TechnologieAllianz e.V.
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There are currently great hopes for solid-state batteries. They contain no liquid parts that could leak or catch fire. For this reason, they do not require cooling and are considered to be much safer, more reliable, and longer lasting than traditional lithium-ion batteries. Jülich scientists have now introduced a new concept that allows currents up to ten times greater during charging and discharging than previously described in the literature. The improvement was achieved by a “clever” choice of materials with a focus on consistently good compatibility. All components were made from phosphate compounds, which are well matched both chemically and mechanically.
The low current is considered one of the biggest hurdles in the development of solid-state batteries. It is the reason why the batteries take a relatively long...
New design tool automatically creates nanostructure 3D-print templates for user-given colors
Scientists present work at prestigious SIGGRAPH conference
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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....
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