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!
New malaria analysis method reveals disease severity in minutes
14.08.2017 | University of British Columbia
New type of blood cells work as indicators of autoimmunity
14.08.2017 | Instituto de Medicina Molecular
Whether you call it effervescent, fizzy, or sparkling, carbonated water is making a comeback as a beverage. Aside from quenching thirst, researchers at the University of Illinois at Urbana-Champaign have discovered a new use for these "bubbly" concoctions that will have major impact on the manufacturer of the world's thinnest, flattest, and one most useful materials -- graphene.
As graphene's popularity grows as an advanced "wonder" material, the speed and quality at which it can be manufactured will be paramount. With that in mind,...
Physicists at the University of Bonn have managed to create optical hollows and more complex patterns into which the light of a Bose-Einstein condensate flows. The creation of such highly low-loss structures for light is a prerequisite for complex light circuits, such as for quantum information processing for a new generation of computers. The researchers are now presenting their results in the journal Nature Photonics.
Light particles (photons) occur as tiny, indivisible portions. Many thousands of these light portions can be merged to form a single super-photon if they are...
For the first time, scientists have shown that circular RNA is linked to brain function. When a RNA molecule called Cdr1as was deleted from the genome of mice, the animals had problems filtering out unnecessary information – like patients suffering from neuropsychiatric disorders.
While hundreds of circular RNAs (circRNAs) are abundant in mammalian brains, one big question has remained unanswered: What are they actually good for? In the...
An experimental small satellite has successfully collected and delivered data on a key measurement for predicting changes in Earth's climate.
The Radiometer Assessment using Vertically Aligned Nanotubes (RAVAN) CubeSat was launched into low-Earth orbit on Nov. 11, 2016, in order to test new...
A study led by scientists of the Max Planck Institute for the Structure and Dynamics of Matter (MPSD) at the Center for Free-Electron Laser Science in Hamburg presents evidence of the coexistence of superconductivity and “charge-density-waves” in compounds of the poorly-studied family of bismuthates. This observation opens up new perspectives for a deeper understanding of the phenomenon of high-temperature superconductivity, a topic which is at the core of condensed matter research since more than 30 years. The paper by Nicoletti et al has been published in the PNAS.
Since the beginning of the 20th century, superconductivity had been observed in some metals at temperatures only a few degrees above the absolute zero (minus...
16.08.2017 | Event News
04.08.2017 | Event News
26.07.2017 | Event News
18.08.2017 | Life Sciences
18.08.2017 | Physics and Astronomy
18.08.2017 | Materials Sciences