A potential way to treat muscular dystrophy directly targets muscle repair instead of the underlying genetic defect that usually leads to the disease.
Muscular dystrophies are a group of muscle diseases characterized by skeletal muscle wasting and weakness. Mutations in certain proteins, most commonly the protein dystrophin, cause muscular dystrophy in humans and also in mice.
A University of Michigan team led by cell biologist Haoxing Xu, discovered that mice missing a critical calcium channel inside the cell, called TRPML1, showed similar muscle defects as those present in muscular dystrophy patients. Though these mice did not have the defect in dystrophin, they still developed muscular dystrophy-like muscle characteristics.
When researchers increased the activity of the calcium channel in the muscular dystrophic mice, it improved muscle membrane repair and restored muscle function.
"The hope is that the same calcium channel will work in people with muscular dystrophy," Xu said.
The long-term plan is to develop clinical trials of a drug that would provide the extra activity of TRPML1.
The findings are scheduled for advance online publication Sept. 14 in Nature Medicine. Xiping Cheng, U-M Department of Molecular, Cellular, and Developmental Biology, is first author on the paper.
While the treatment in mice using a gene therapy approach involved a viral carrier to insert extra TRPML1 channels in muscle, Xu says his team has recently discovered a drug that can activate this calcium channel. This drug might provide a similar boost to muscle membrane repair in human muscular dystrophy patients, but this requires further test in the laboratory.
This research was supported by grants from National Institute of Arthritis and Musculoskeletal and Skin Diseases and National Institute of Neurological Disorders and Stroke.
More on Haoxing Xu: http://bit.ly/1xIDxux
Laura Bailey | Eurek Alert!
A novel synthetic antibody enables conditional “protein knockdown” in vertebrates
20.08.2018 | Technische Universität Dresden
Climate Impact Research in Hannover: Small Plants against Large Waves
17.08.2018 | Leibniz Universität Hannover
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
Most of the objects we see are colored by pigments, but using pigments has disadvantages: such colors can fade, industrial pigments are often toxic, and...
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....
17.08.2018 | Event News
08.08.2018 | Event News
27.07.2018 | Event News
20.08.2018 | Information Technology
20.08.2018 | Life Sciences
20.08.2018 | Information Technology