Navigation Links
Research shows promise for potential new gene therapy strategy for muscle-wasting diseases

Investigators in The Research Institute at Nationwide Childrens Hospital have identified the role of a protein that could potentially lead to new clinical treatments to combat musculoskeletal diseases, including Duchenne muscular dystrophy (DMD).

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 Childrens 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.


Contact: Pam Barber/Mary Ellen Fiorino
Nationwide Children's Hospital

Related medicine news :

1. Mayo Clinic proceedings highlights research about cardiovascular benefits of omega-3 fatty acids
2. University of Pittsburgh researchers crack code of 3-D structure in key metabolic protein
3. UCF researchers discover a new protein family implicated in inflammatory diseases
4. Size does matter: Researchers adapt drug dosing to body size
5. Genetic research unveils common origins for distinct clinical diagnoses
6. Burroughs Wellcome Fund grants $9.75 million towards translational researchers
7. Researchers Design Promising Cancer Drug
8. Case Western Reserve University researchers identify colorectal cancer gene
9. Human, animal vaccine development goal of hepatitis E virus research
10. On a roll: MIT researchers devise new cell-sorting system
11. Rett syndrome research reveals high fracture risk
Post Your Comments:
(Date:6/26/2016)... ... 2016 , ... Brent Kasmer, a legally blind and certified personal trainer is helping to develop ... The fitness app plans to fix the two major problems leading the fitness industry today:, ... all type program , They don’t eliminate all the reasons people quit their ...
(Date:6/25/2016)... ... June 25, 2016 , ... ... issues and applications at AcademyHealth’s Annual Research Meeting June 26-28, 2016, at the ... several important health care topics including advance care planning, healthcare costs and patient ...
(Date:6/25/2016)... , ... June 25, 2016 , ... The temporary closing of Bruton Memorial Library on ... Observer , brings up a new, often overlooked aspect of head lice: the parasite’s ability ... fumigation is not a common occurrence, but a necessary one in the event that lice ...
(Date:6/25/2016)... ... June 25, 2016 , ... First Choice Emergency Room , the ... Ogunleye, as the Medical Director of its new Mesquite-Samuell Farm facility. , “We ... new Mesquite location,” said Dr. James M. Muzzarelli, Executive Medical Director of First Choice ...
(Date:6/25/2016)... ... June 25, 2016 , ... Dr. Calvin ... injury. Recently, he has implemented orthobiologic procedures as a method for treating his ... of the first doctors to perform the treatment. Orthobiologics are substances that orthopaedic ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... , June 23, 2016 Research ... Devices Global Market - Forecast to 2022" report to ... the treatment method for the patients with kidney failure, it ... excess fluid from the patient,s blood and thus the treatment ... potassium and chloride in balance. Increasing number ...
(Date:6/23/2016)... 2016 Research and Markets has ... by Type (Organic Chemical (Sugar, Petrochemical, Glycerin), Inorganic Chemical), ... Parenteral) - Global Forecast to 2021" report to ... The global pharmaceutical excipients market is projected to reach ... 6.1% in the forecast period 2016 to 2021. ...
(Date:6/23/2016)... Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that it ... (procalcitonin) assay as a dedicated testing solution for people ... Roche is the first IVD company in the U.S ... assessment and management. PCT is a sepsis-specific ... blood can aid clinicians in assessing the risk of ...
Breaking Medicine Technology: