Results of these studies appear in the March 11, 2008 issue of the Proceedings of the National Academy of Sciences.
These studies, led by Brian Kaspar, PhD, a principal investigator in the Center for Gene Therapy at The Research Institute and an assistant professor of Pediatrics at The Ohio State University, focus on a protein called follistatin (FS). Using a single injection, gene-delivery strategy involving FS, investigators treated the hind leg muscles of mice. Results showed increased muscle size and strength, quadruple that of mice treated with proteins other than FS. The muscle enhancements were shown to be well-tolerated for more than two years.
According to Dr. Kaspar, increased muscle mass and strength were also evident when this strategy was tested using a model of DMD. Apart from the injected hind leg muscles, strengthening effects were also shown in the triceps. In addition, fibrosis, abnormal formation of scar tissue and a hallmark of muscular dystrophy, was decreased in FS-treated animals.
“We believe this new FS strategy may be more powerful than other strategies due to its additional effects, including its ability to reduce inflammation,” said Dr. Kaspar.
The strategy showed no negative effects on the heart or reproductive ability of either males or females. The results were also replicated in older animals, suggesting that this strategy could be useful in developing clinical treatments for older DMD patients.
“This research provides evidence of multiple potential treatment applications for muscle diseases including, but not limited to, muscular dystrophy,” said Jerry Mendell, MD, director of the Center for Gene Therapy at The Research Institute, a co-author on the study, and professor of Pediatrics in Neurology and Pathology at The Ohio State University. “These results offer promise for treatment of potentially any muscle-wasting disease, including muscle weakness due to other illnesses, aging, and inflammatory diseases such as polymyositis. Our next step is to pursue clinical trials.”
The Research Institute at Nationwide Children’s Hospital has a patent pending on the FS technique due to the major role it may play for muscular dystrophy treatment and other muscle-wasting diseases.
Pam Barber | EurekAlert!
Fueling Replication - how herpes viruses get to cellular DNA building blocks
24.09.2019 | Universitätsklinikum Tübingen
Study points to new drug target in fight against cancer
19.09.2019 | Rice University
For applications such as light-emitting diodes or solar cells, organic materials are nowadays in the focus of research. These organic molecules could be a promising alternative to currently used semiconductors such as silicon or germanium and are used in OLED displays. A major problem is that in many organic semiconductors the flow of electricity is hampered by microscopic defects. Scientists around Dr. Gert-Jan Wetzelaer and Dr. Denis Andrienko of the Max-Planck-Institute for Polymer Research have now investigated how organic semiconductors can be designed such that the electric conduction is not influenced by these defects.
The basic principle of the first light bulb, invented by Thomas Edison in the 19th century, was quite simple: Electrons – negatively charged particles – flow...
How long the battery of your phone or computer lasts depends on how many lithium ions can be stored in the battery's negative electrode material. If the battery runs out of these ions, it can't generate an electrical current to run a device and ultimately fails.
Materials with a higher lithium ion storage capacity are either too heavy or the wrong shape to replace graphite, the electrode material currently used in...
To process information, photons must interact. However, these tiny packets of light want nothing to do with each other, each passing by without altering the...
Researchers from the Department of Atomically Resolved Dynamics of the Max Planck Institute for the Structure and Dynamics of Matter (MPSD) at the Center for Free-Electron Laser Science in Hamburg, the University of Hamburg and the European Molecular Biology Laboratory (EMBL) outstation in the city have developed a new method to watch biomolecules at work. This method dramatically simplifies starting enzymatic reactions by mixing a cocktail of small amounts of liquids with protein crystals. Determination of the protein structures at different times after mixing can be assembled into a time-lapse sequence that shows the molecular foundations of biology.
The functions of biomolecules are determined by their motions and structural changes. Yet it is a formidable challenge to understand these dynamic motions.
At the International Symposium on Automotive Lighting 2019 (ISAL) in Darmstadt from September 23 to 25, 2019, the Fraunhofer Institute for Organic Electronics, Electron Beam and Plasma Technology FEP, a provider of research and development services in the field of organic electronics, will present OLED light strips of any length with additional functionalities for the first time at booth no. 37.
Almost everyone is familiar with light strips for interior design. LED strips are available by the metre in DIY stores around the corner and are just as often...
19.09.2019 | Event News
10.09.2019 | Event News
04.09.2019 | Event News
24.09.2019 | Life Sciences
24.09.2019 | Life Sciences
24.09.2019 | Materials Sciences