Stimulating the production of utrophin with heregulin improves the quality of muscle tissue in mdx mice (right). Credit: Tejvir S. Khuranal/Proceedings of the National Academy of Sciences.
Researchers at the University of Pennsylvania School of Medicine report a novel strategy for stimulating the production of utrophin – an important muscle protein in young mice – for muscular dystrophy therapy. The investigators gave mdx mice (the mouse model for Duchenne’s muscular dystrophy) heregulin, a small molecule to turn on the production of utrophin in their muscles. Utrophin improved muscle function in the mdx mice. "Our strategy boosts the levels of an existing gene using pre-existing cellular machinery rather than having to deliver a gene via gene therapy," says lead author Tejvir S. Khurana, MD, PhD, Assistant Professor of Physiology & Member of the Pennsylvania Muscle Institute.
They detected an approximately threefold increase of utrophin levels over control mdx mice. "This is the level at which one starts seeing a therapeutic affect, as measured in lab tests with mouse muscles," says Khurana. The researchers noted an improvement in the quality of mouse muscle tissue, the biomechanical properties of muscles, and biochemical indices of dystrophy in the muscles.
In patients with Duchenne’s muscular dystrophy (DMD), the gene to make the protein dystrophin is missing, which results in the muscle wasting that is associated with the disease. The progressive muscle wasting begins in early childhood and typically leads to death in the twenties. "The gene for utrophin is already in the body, so by giving a small peptide to stimulate its production, we’re bypassing the need for dystrophin by cranking up the levels of utrophin," explains Khurana. This research appears in the September 21 issue of the Proceedings of the National Academy of Sciences.
Karen Kreeger | EurekAlert!
Millions through license revenues
27.04.2017 | Rheinische Friedrich-Wilhelms-Universität Bonn
New High-Performance Center Translational Medical Engineering
26.04.2017 | Fraunhofer ITEM
More and more automobile companies are focusing on body parts made of carbon fiber reinforced plastics (CFRP). However, manufacturing and repair costs must be further reduced in order to make CFRP more economical in use. Together with the Volkswagen AG and five other partners in the project HolQueSt 3D, the Laser Zentrum Hannover e.V. (LZH) has developed laser processes for the automatic trimming, drilling and repair of three-dimensional components.
Automated manufacturing processes are the basis for ultimately establishing the series production of CFRP components. In the project HolQueSt 3D, the LZH has...
Reflecting the structure of composites found in nature and the ancient world, researchers at the University of Illinois at Urbana-Champaign have synthesized thin carbon nanotube (CNT) textiles that exhibit both high electrical conductivity and a level of toughness that is about fifty times higher than copper films, currently used in electronics.
"The structural robustness of thin metal films has significant importance for the reliable operation of smart skin and flexible electronics including...
The nearby, giant radio galaxy M87 hosts a supermassive black hole (BH) and is well-known for its bright jet dominating the spectrum over ten orders of magnitude in frequency. Due to its proximity, jet prominence, and the large black hole mass, M87 is the best laboratory for investigating the formation, acceleration, and collimation of relativistic jets. A research team led by Silke Britzen from the Max Planck Institute for Radio Astronomy in Bonn, Germany, has found strong indication for turbulent processes connecting the accretion disk and the jet of that galaxy providing insights into the longstanding problem of the origin of astrophysical jets.
Supermassive black holes form some of the most enigmatic phenomena in astrophysics. Their enormous energy output is supposed to be generated by the...
The probability to find a certain number of photons inside a laser pulse usually corresponds to a classical distribution of independent events, the so-called...
Microprocessors based on atomically thin materials hold the promise of the evolution of traditional processors as well as new applications in the field of flexible electronics. Now, a TU Wien research team led by Thomas Müller has made a breakthrough in this field as part of an ongoing research project.
Two-dimensional materials, or 2D materials for short, are extremely versatile, although – or often more precisely because – they are made up of just one or a...
28.04.2017 | Event News
20.04.2017 | Event News
18.04.2017 | Event News
28.04.2017 | Medical Engineering
28.04.2017 | Earth Sciences
28.04.2017 | Life Sciences