Navigation Links
Human protein improves muscle function of muscular dystrophy mice
Date:12/27/2010

PROVIDENCE, R.I. [Brown University] A novel potential therapy based on a natural human protein significantly slows muscle damage and improves function in mice who have the same genetic mutation as boys with the most common form of muscular dystrophy, according to a paper published online Dec. 27 in the Proceedings of the National Academy of Sciences.

Duchenne Muscular Dystrophy is a fatal genetic mutation in about one of every 3,500 boys. They are unable to produce a protein called dystrophin that keeps muscles strong. By eight years of age, the boys begin to have trouble walking. By their teens they are often in wheelchairs, and by their 20s muscle function is so degraded that they die.

"This is all aimed at getting a therapy that will meaningfully improve the condition of patients," said Justin Fallon, professor of neuroscience at Brown University and the senior author of the paper. "This is an important step along that path."

This fall, the startup company Tivorsan Pharmaceuticals licensed rights from Brown to the key protein, biglycan, hoping to bring the potential therapy through clinical trials.

Biglycan restores the muscle-strengthening presence of a protein called utrophin, which is normally prevalent only in very young children. Utrophin still exists in adults, but in fewer places and not where it can help muscular dystrophy sufferers who cannot produce dystrophin, which keeps adult muscles strong.

Encouraging experiments

In experiments described in the paper, Fallon's team showed that biglycan delivered to the bloodstream draws utrophin to the cellular membranes of muscle cells. Much as utrophin does when it is present in fetuses, infants and toddlers, the protein works to help the cells build and retain their strength.

In one experiment, Fallon's team found a 50-percent reduction in "centrally nucleated" fibers in the muscle tissue of biglycan treated mice compared to untreated mice. Biologists recognize the fibers as indicators of recent tissue damage and repair, so a reduction in them suggests that the muscle tissue is suffering less damage.

The team also subjected mouse muscles to a standardized stress test where they are simultaneously stretched and caused to contract. The test ultimately weakens even healthy muscle, but in the tests conducted by researchers and co-authors from the University of Pennsylvania, the muscles of muscular dystrophy mice treated with biglycan lost their strength 30 percent more slowly than similar mice who were untreated.

In more recent tests using an improved formulation of biglycan, Fallon said, the team has seen that figure rise to 50 percent in some muscles, meaning that mice treated with biglycan are holding on to more of their function for a longer time.

Fallon said the effects of treatment with biglycan lasted through months of testing. Several basic tests for side effects during that time frame, such as kidney and liver function, did not indicate any harm from the therapy.

With the efficacy of biglycan apparent in the mouse model of Duchenne Muscular Dystrophy, Fallon is eager to see if it can improve the lives of thousands of children.

"The next big step is testing in humans," Fallon said.


'/>"/>

Contact: David Orenstein
david_orenstein@brown.edu
401-863-1862
Brown University
Source:Eurekalert

Related medicine news :

1. Actor and Humanitarian Harrison Ford Assists Operation Smiles Relief Efforts in Haiti
2. Human Dental Technology Leads to Breakthroughs for Pet Dental Health at Sergeants Pet Care Products, Inc.
3. Scripps Health Begins Pioneering Study of Human Tumor Sequencing in Cancer Patients
4. New drug for kidney transplant recipients effective in humans
5. Agility Consulting Partners with pan (Performance Assessment Network) to Bring Custom Private Label Assessment Capability to Human Resource Professionals and Consultants
6. Dolphins health shed light on human and ocean health
7. Dolphins could be ideal model to study human cervical cancer, UF veterinarians say
8. Dolphins May Warn of Health Risks in Humans
9. Inadequate access to opioid-based pain relief is a human rights issue for cancer patients
10. Sociologist expands US conversation on human rights
11. State of Oregon Department of Human Services Selects Netsmart Technologies for Comprehensive Behavioral Healthcare Integration Project
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/27/2016)... (PRWEB) , ... June 27, 2016 , ... "FCPX editors ... customizable inside of Final Cut Pro X," said Christina Austin - CEO of Pixel ... style. Final Cut Pro X users can now reveal the media of ...
(Date:6/27/2016)... ... June 27, 2016 , ... TopConsumerReviews.com recently awarded their highest five-star rating ... Millions of individuals in the United States and Canada wear eyeglasses. Once considered to ... correct vision and make a fashion statement. Even celebrities use glasses as a way ...
(Date:6/26/2016)... Carolina (PRWEB) , ... June 26, 2016 , ... ... of a new product that was developed to enhance the health of felines. The ... centuries. , The two main herbs in the PawPaws Cat Kidney Support ...
(Date:6/25/2016)... ... June 25, 2016 , ... Austin residents seeking Mohs surgery services, can ... Surgery and to Dr. Russell Peckham for medical and surgical dermatology. , Dr. Dorsey ... cancer. The selective fellowship in Mohs Micrographic Surgery completed by Dr. Dorsey was under ...
(Date:6/25/2016)... ... June 25, 2016 , ... As a lifelong Southern Californian, ... and his M.D from the David Geffen School of Medicine at UCLA. He trained ... Angeles to complete his fellowship in hematology/oncology at the UCLA-Olive View-Cedars Sinai program where ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... 23, 2016 Research and Markets has ... Market Analysis 2016 - Forecast to 2022" report to ... report contains up to date financial data derived from varied ... major trends with potential impact on the market during the ... segmentation which comprises of sub markets, regional and country level ...
(Date:6/23/2016)... Ill. and INDIANAPOLIS , ... students receiving a Lilly Diabetes Tomorrow,s Leaders Scholarship is ... 2016 scholarship winners, announced today online at www.diabetesscholars.org ... let type 1 diabetes stand in the way of ... has supported the Foundation,s scholarship program since 2012, and ...
(Date:6/23/2016)... Research and Markets has announced the addition ... States, China, Japan, Brazil, United Kingdom, Germany, France, Italy, ... Surgical Procedure Volumes: Global ... surgical procedure volume data in a geographic context. The ... of growth drivers and inhibitors, including world population growth, ...
Breaking Medicine Technology: