Navigation Links
Research shows promise for potential new gene therapy strategy for muscle-wasting diseases
Date:3/10/2008

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
614-355-0495
Nationwide Children's Hospital
Source:Eurekalert

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:
*Name:
*Comment:
*Email:
(Date:2/7/2016)... ... February 07, 2016 , ... HealthSmart Holdings, Inc. ... organizations with the tools and information to lower the costs, and increase the ... cost of providing employee healthcare benefits by as much as 22%:, + ...
(Date:2/6/2016)... ... February 06, 2016 , ... ... , MEDIA ADVISORY: 5000 PERIOPERATIVE NURSES EXPECTED AT AORN SURGICAL CONFERENCE & ... nurses in the world with an estimated 5000 perioperative nurses in attendance ...
(Date:2/6/2016)... ... 2016 , ... With the FCPX LUT: Summer pack from Pixel ... A LUT is a Lookup Table that contains a mathematical formula for modifying an ... table. By manipulating each pixel, LUT's can change each color range differently, it gives ...
(Date:2/6/2016)... ... February 06, 2016 , ... Research ... treatment helps to reduce the frequency and level of relapse. , ... Healthy Identity and Purpose,” will explore the critical tasks of the recovery phase ...
(Date:2/5/2016)... , ... February 05, 2016 , ... Love is in ... in a variety of colors, assortments and packaging. This staple for Valentine’s Day is ... Kullen location. , For Valentine’s Day, not only are long-stem roses available, but ...
Breaking Medicine News(10 mins):
(Date:2/8/2016)... ORLANDO, Fla. , Feb. 8, 2016 ... and marketing high performance apparel for healthcare and other ... Bold Chief Executive Officer. He replaces Dale ... and remains Chairman. Mr. Bold is also joining Vestagen,s ... in a new class of active barrier technologies that ...
(Date:2/8/2016)... Corporation ("Unilife" or "Company") (NASDAQ: UNIS ; ASX: UNS), ... announced that it will release its financial results for the ... close on February 9, 2016.  At this time, the Company ... results.    About Unilife Corporation --> ... Unilife Corporation UNIS / ASX: UNS) ...
(Date:2/8/2016)... Feb. 8, 2016  Astellas Pharma Inc. President and Chief ... of James Robinson as president, Americas Operations, for ... North and South America , effective April ... representing the commercial organization in the United States ... Masao Yoshida , who is retiring in June ...
Breaking Medicine Technology: